Simultaneous Measurement and Responsive Treatment (SMART01): A Feasibility Study of the SynerG TM CGM sensor Integrated with the Niia Essential Insulin Patch Pump System in Persons with Type 1 Diabetes.

Start Date24 Mar 2025

Sponsor / Collaborator-

NL-OMON57171

/ SuspendedNot Applicable

Phoenix: Clinical evaluation of the Medtronic Implantable Insulin Pump System (MIIPS 2020) in adult subjects with Type 1 Diabetes - Phoenix

Start Date06 Nov 2024

Sponsor / Collaborator-

100 Clinical Results associated with NNC0268-0965

100 Translational Medicine associated with NNC0268-0965

100 Patents (Medical) associated with NNC0268-0965

122,013

Literatures (Medical) associated with NNC0268-0965

31 Dec 2025·ANNALS OF MEDICINE

Bioengineering and omics approaches for Type 1 diabetes practical research: advancements and constraints

Review

Author: Li, Ling ; An, Zhenmei ; Peng, Yihua ; Peng, Xi ; Zhou, Guangju

Insulin dependency arises from autoimmunity that targets the β cells of the pancreas, resulting in Type 1 diabetes (T1D). Despite the fact that T1D patients require insulin for survival, insulin does not provide a cure for this disease or prevent its complications. Despite extensive genetic, molecular, and cellular research on T1D over the years, the translation of this understanding into effective clinical therapies continues to pose a significant obstacle. It is therefore difficult to develop effective clinical treatment strategies without a thorough understanding of disease pathophysiology. Pancreatic tissue bioengineering models of human T1D offer a valuable approach to examining and controlling islet function while tackling various facets of the condition. And in recent years, due to advances in high-throughput omics analysis, the genotypic and molecular profiles of T1D have become finer tuned. The present article will examine recent progress in these areas, along with their utilization and constraints in the realm of T1D.

31 Dec 2025·Journal of Pharmaceutical Policy and Practice

Emergency medicine pharmacists’ interventions in the tertiary hospitals’ emergency departments in Malaysia

Article

Author: Teh, Lih Jiuan ; Tan, Shirlyn ; Tan, Sherene Su Ann ; Bhuvanendran Pillai, Anaanthan ; Sia, Rosalind Guen Lin ; Guee, Xin Nee ; Selvaratanam, Manimegahlai ; Koh, Hock Peng ; Koo, Yee Ping ; Wong, Wai Kit ; Chen, Mary Siew Yng ; Ab Jalal, Haliza

Background:

The emergency medicine (EM) pharmacist is an integrated part of the Emergency Department (ED) interdisciplinary team in many countries, including Malaysia. The presence of EM pharmacists in the ED has positively impacted patient outcomes. Data on EM pharmacists' interventions is scarce in the Asian region. In Malaysia, data on interventions done by EM pharmacists in the EDs was unavailable. This study aimed to assess the type of interventions done by EM pharmacists in the ED of tertiary public hospitals in Malaysia.

Methods:

This cross-sectional, multicenter study involved EM pharmacists from 14 tertiary hospitals in Malaysia. All accepted interventions done by EM pharmacists in the ED for patients admitted to the Red (critical) and Yellow (semi-critical) zones from January to June 2022 were extracted from the Clinical Pharmacy Report Form. All data were analyzed descriptively.

Results:

The EM pharmacists documented 1659 accepted interventions on 1584 patients during the study period. Inappropriate regimens (n = 1117, 67.3%) and incomplete prescriptions (n = 339, 20.4%) were the main categories of accepted interventions in ED. Inappropriate drug (n = 574, 34.6%), dose (n = 292, 17.6%), and frequency (n = 176, 10.6%) were the top three subcategory interventions documented under inappropriate regimens. Antimicrobials, antihypertensives, and proton pump inhibitors were the commonest drug intervened under the categories of inappropriate drug intervention. There were 272 (16.4%) accepted interventions on high-alert medications (HAMs). Insulin, enoxaparin, and noradrenaline were the most intervened HAMs.

Conclusion:

Inappropriate treatment regimens were the most common intervention category done by EM pharmacists in Malaysia. The significant number of interventions done by EM pharmacists demonstrated the importance of EM pharmacists as integral members of the EM team. This data can help improve the quality of clinical pharmacy services in the ED and is important for the future expansion of clinical pharmacy services in all EDs across Malaysia, neighbouring countries, and other developing countries.

31 Dec 2025·Journal of Pharmaceutical Policy and Practice

Effect of statins and antihyperglycemics on chronic kidney disease in patients with type 2 diabetes mellitus: a retrospective cohort study with a 12-year follow-up

Article

Author: El-Dahiyat, Faris ; Mohammed, Khuloud Jamal ; Al Jaberi, Asma Khaled ; Al Jawamis, Dina Farhan ; Shahwan, Moyad ; Jairoun, Ammar Abdulrahman ; Ping, Chong Chee ; Dawoud, Tasnim ; Ibrahim, Baharudin

Background:

Chronic Kidney Disease (CKD) represents a significant worldwide health challenge, with far-reaching implications for both patients and healthcare systems. This study aimed to identify the incidence of CKD at stages 3-5, analyzed the impact of statin and other antihyperglycemic interventions, on the CKD progression in individuals with T2DM.

Methods:

This was a single-center retrospective cohort study based on data derived from electronic medical records (EMR) of UAE populations with diabetes mellitus, registered at outpatient clinics at Tawam Hospital in Al Ain, UAE, between January 2011 and December 2021. T2DM patients aged ≥ 18 years who had serum HbA1c level ≥ 6.5% and using one of the statin therapies were inclusion criteria. Patients with T1DM, who had undergone permanent renal replacement therapy, with under 1 year of follow-up and missing or incomplete data were excluded from the study. The collected data encompassed socio-demographics, detailed medical history, anthropometric measurements, laboratory analyses, clinical parameters, disease characteristics, and medications.

Results:

Our study included a cohort of 1,003 individuals. We observed 388 subjects developed CKD stages 3-5 across an average monitoring duration of 11.7 years. This resulted in a cumulative incidence of 38.7%, translating to an incidence rate of 38 cases per 1000 person-years. There was a statistically significant difference in the cumulative incidence of CKD stages 3 ± 5 according to statin therapy (P = 0.047). High intensity statin users are more likely to develop a CKD stage 3-5 compared to low/moderate intensity users and to no statin users respectively (44.3% vs 37.9%), (44.3% vs 30.9%). Conversely, the use of Biguanides was associated with a decreased probability of CKD progression (37.9% vs. 52.8%; P = 0.001), whereas Insulin users demonstrated a heightened risk (54.2% vs. 34.1%; P < 0.001).

Conclusion:

The findings emphasise the pivotal role of personalised treatment strategies, particularly concerning statin therapy and other medications, in populations at high risk.

786

News (Medical) associated with NNC0268-0965

10 Jun 2025

·www.businesswire.com

Navitus Drug Trend Report: Keeping Costs Low for Plans and Members in an Era of Accelerating Inflation

MADISON, Wis.--(BUSINESS WIRE)--Navitus, the nation’s first transparent, pass-through pharmacy benefit manager (PBM), released its ninth annual Drug Trend Report today, revealing how commercial plan sponsors saved millions in prescription costs while maintaining high-quality member care. 30% of Navitus clients spent less in 2024 than in the previous year, while overall client book of business drug cost trend was managed to 7%. The data shows that medications such as glucagon-like peptide-1 receptor agonists (GLP-1 RAs) are creating unprecedented financial pressure on plan sponsors as utilization for diabetes treatment increased 26% year-over-year. Overall health spending in the United States is nearly $4.9 trillion and projected to increase nearly 6% annually over the next decade.1 A subset of this, prescription drug spending, was estimated to be $487 billion in 2024, an industry-wide 11.4% increase over the previous year, compared to 7% for Navitus clients. While the industry faces these escalating cost pressures, our latest Drug Trend Report highlights how Navitus continues to outperform industry trends and save clients money in this challenging environment, including companies such as Progressive Insurance. “Navitus stands out for its commitment to transparency and managing to lowest net cost, which has significantly benefited Progressive and its members. From the outset, their 100% pass-through model has ensured that we receive the full value of manufacturer rebates and discounts, directly translating to reduced pharmacy costs,” said Heidi Minter, Data Analyst, Benefits & Design Services, Progressive Insurance. The Navitus Drug Trend Report revealed three critical trends influencing the broader industry: 26% increase in GLP-1 RAs use for treatment of type 2 diabetes. While delivering significant clinical benefits, these medications are contributing substantially to pharmaceutical cost increases for benefit plans: Net trend in the diabetes category increased 8.5%, driven by growth in both the use of GLP-1 RAs, including Ozempic, Mounjaro and Trulicity, and a 16% increase in use of sodium-glucose cotransporter-2 inhibitors (SGLT-2s), including Farxiga and Jardiance. The prevalence and cost of GLP-1 RAs and SLGT-2s resulted in an overall unit cost increase of 3.0% for the diabetes category, in spite of the net cost decrease of more than 28% for insulin in 2024. Biosimilars delivered $315 million in savings with a 60% reduction in Humira costs. Targeted immunomodulators (TIMs) treat a wide range of autoimmune disorders, from rheumatoid arthritis and psoriasis to inflammatory bowel diseases. The launch of Humira biosimilar alternatives in 2023 significantly changed the landscape of the category as these clinically-equivalent, much lower cost options were added to formulary. After adding biosimilars to formulary, Navitus took a decisive step to lower costs further by removing Humira from formulary in June 2024. This led to key results including: Over $315 million in upfront cost savings. 60% reduction in net costs per claim. Specialty medication utilization increased 12%. Approximately 75% of new drug approvals have been for specialty or medical specialty agents, and that trend is expected to continue in the next two years. Key factors included: Oncology net trend increased more than 13%, driven by both higher utilization and unit cost: Utilization of newer breast cancer treatments (Kisqali, Verzenio) increased 20%, driving overall cost. In specialty dermatology, Dupixent (dupilumab) emerged as the most rapidly growing drug of 2024. The category saw a net trend increase of more than 45% and Dupixent represented 96% of category cost and utilization. While increased use of newer, more expensive agents like Kesimpta created upward pressure within the multiple sclerosis category, generics represented more than 50% of prescriptions filled at 10% of the cost. “Our annual Drug Trend Report confirms we were able to control year-over-year costs for commercial clients - both large and small - and deliver savings greater than the estimated industry averages,” said Sharon Faust, PharmD, MBA, CSP, Chief Pharmacy Officer, Navitus Health Solutions. “Deploying generic-first strategies, facilitating adoption of new biosimilars, ensuring 100% pass-through of negotiated rebates and discounts, and supporting clinically appropriate prescribing, utilization and formulary management remain core to our focus.” Access the Navitus Drug Trend Report executive summary here: www.navitus.com/DTR2024 Methodology: The Navitus drug trend is calculated by comparing the net total cost per-member per-month (PMPM) for 2024 to that for 2023. Net cost PMPM represents full-year (Q1-Q4) data for total member copays and plan paid amounts minus manufacturer rebates and fees. This value is divided by the total number of members and by 12 months of the year. The data represents employer plan sponsors and health plans. In order to be included organizations must have been with Navitus for two full calendar years. *Due to varying coverage decisions by clients, GLP-1s for weight loss were not included in the Drug Trend Report, yet they still warrant thoughtful consideration and conversation in the overall picture of trend and future decisions. The DTR only represents prescriptions under the pharmacy benefit. 40% of Rx spend is through the medical benefit and warrants careful consideration by plans. Sources: 1 American Medical Association, Trends in Healthcare Spending. https://www.ama-assn.org/about/research/trends-health-care-spending#:~:text=Health%20spending%20in%20the%20U.S.,%25)%20and%202021%20(18.3%25). Updated April 17, 2025. Accessed April 23, 2025. 2 IQVIA, Understanding the Use of Medicines in the U.S. 2025: Evolving Standards of Care, Patient Access, and Spending. https://www.iqvia.com/insights/the-iqvia-institute/reports-and-publications/reports/understanding-the-use-of-medicines-in-the-us-2025. Published April 2025, Accessed May 15, 2025. About Navitus Navitus remains the nation’s first transparent, pass-through pharmacy benefit manager (PBM). It uniquely brings clarity to drug pricing and takes costs out of the drug supply chain. Unlike traditional PBMs that generate profit by retaining an undisclosed portion of rebates and discounts negotiated with drug manufacturers and pharmacies, Navitus passes along the complete savings to clients, enabling them to make medication more affordable for their members. Navitus was established more than 20 years ago by Navitus Health Solutions, LLC, a pioneering pharmacy solutions company. The organization delivers a range of services through portfolio brands including Navitus, Lumicera, Archimedes. Owned by SSM Health and Costco, Navitus Health Solutions serves over 18 million lives across 800 clients including employers, unions, government plans, payors and health systems. For more information, please visit www.navitus.com.

06 Jun 2025

·www.pharmaceutical-technology.com

SCHOTT Pharma invests in RTU cartridge capacity expansion

The new facility in Hungary will create 100 local jobs. Credit: SCHOTT Pharma. Drug containment and delivery solutions provider SCHOTT Pharma has announced an investment of more than €100m ($114m) to expand its manufacturing capacity for sterile ready-to-use (RTU) cartridges at its site in Lukácsháza, Hungary. The new facility will create 100 jobs. RTU cartridges are essential for storing glucagon-like peptide-1 drugs, biologics, insulin and hormone therapies used for treating diabetes, obesity and other immunological conditions. It will further extend the site’s manufacturing capabilities for high-value solutions. SCHOTT Pharma plans to implement a fully integrated and automated production process with minimal manual intervention, ensuring the delivery of sterile products. The facility will also feature a new washing line and a steam sterilisation process for cartridges, aimed at decreasing the environmental impact of the operations. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence SCHOTT Pharma CEO Andreas Reisse stated: “To meet growing demand, we are expanding our capabilities and presence in the diabetes and obesity fields. That is why we are investing more than €100m in our plant in Hungary.” In June 2024, the company opened a new facility at the same site, which has already begun producing high-quality prefillable glass syringes. With an additional 120 employees, the site has increased its production capacity to meet the growing demand for these critical components in the healthcare industry. Lukácsháza site manager Eva Szabó stated: “We would like to express our sincerest gratitude to the Ministry of Foreign Affairs and Trade for its support of these two expansion projects. “Lukácsháza is not only an important location for ensuring supply security to customers in the region but also makes an important contribution to SCHOTT Pharma’s global growth strategy.” Lukácsháza will become the second SCHOTT Pharma site, after St Gallen in Switzerland, for sterile cartridge manufacturing. The company’s portfolio includes a variety of delivery systems and drug containment solutions for injectable drugs, such as prefillable glass and polymer syringes, cartridges, ampoules and vials. Pharmaceutical Technology Excellence Awards - Have you nominated? Nominations are now open for the prestigious Pharmaceutical Technology Excellence Awards - one of the industry's most recognised programmes celebrating innovation, leadership, and impact . This is your chance to showcase your achievements, highlight industry advancements , and gain global recognition . Don't miss the opportunity to be honoured among the best - submit your nomination today! Nominate Now

05 Jun 2025

·www.prnewswire.com

Friedreich's Ataxia Market Poised for Rapid Expansion During the Forecast Period (2025-2034) as Drug Pipeline Gains Momentum | DelveInsight

The dynamics of the Friedreich's ataxia market are anticipated to change as companies across the globe are thoroughly working toward the development of new drug therapy options to treat this disease. Key products such as RT001 (Retrotope), Vatiquinone (PTC Therapeutics), and Leriglitazone (Minoryx Therapeutics) might influence the market size during the forecast period. LAS VEGAS, June 5, 2025 /PRNewswire/ -- DelveInsight's Friedreich's Ataxia Market Insights report includes a comprehensive understanding of current treatment practices, Friedreich's ataxia emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Friedreich's Ataxia Market Report According to DelveInsight's analysis, the market size for Friedreich's ataxia is anticipated to surge at a significant rate by 2034. The United States is expected to account for the highest market size of Friedreich's ataxia, of the total market size in 7MM in comparison to the other major markets, i.e., EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan. The prevalence of Friedreich's ataxia in the United States has been gradually increasing, reflecting greater awareness and improved diagnostic capabilities. Advances in genetic testing have allowed for earlier and more accurate identification of the condition, contributing to higher reported cases. Additionally, increased access to healthcare may facilitate more comprehensive assessments and documentation of rare diseases like Friedreich's ataxia. As research progresses, ongoing efforts to understand the genetic and environmental factors influencing this condition will be crucial for developing effective treatments and support systems for affected individuals. Based on a secondary research genetically confirmed Friedreich's ataxia patient has not been found in the Japanese population, and the majority of patients with Friedreich's ataxia-like phenotype may be confirmed as AOA1, AOA2, ataxia with vitamin E deficiency or autosomal-recessive spastic ataxia of Charlevoix-Saguenay if examined by genetic testing. Leading Friedreich's ataxia companies developing emerging therapies, such as Retrotope, PTC Therapeutics, Minoryx Therapeutics, Biogen, Lexeo Therapeutics, Larimar Therapeutics, Design Therapeutics, Solid Biosciences, and others, are developing novel Friedreich's ataxia drugs that can be available in the Friedreich's ataxia market in the coming years. The promising Friedreich's ataxia therapies in the pipeline include RT001, Vatiquinone, Leriglitazone, Omaveloxolone, LX2006, nomlabofusp, SGT-212, and others. Discover which therapies are expected to grab the Friedreich's ataxia therapeutics market share @ Friedreich's Ataxia Market Report Friedreich's Ataxia Market Dynamics The Friedreich's ataxia market dynamics are expected to change in the coming years. Advances in the understanding of Friedreich's ataxia have significantly clarified the role of frataxin and the disease's natural history, providing crucial insights that have guided the development of targeted therapeutic approaches aimed at addressing the underlying pathology. The approval of omaveloxolone, a NF-E2-related factor 2 activator, by the FDA in 2023 and the EU in 2024, marks a milestone in disease-specific treatment. Concurrently, the rising prevalence of Friedreich's ataxia, driven by population growth and improved diagnostic technologies, is expected to fuel market demand. Meanwhile, biomarker development is progressing, with promising tools such as frataxin quantification and imaging biomarkers enhancing clinical trial sensitivity and enabling more precise patient stratification. Furthermore, potential therapies are being investigated for the treatment of Friedreich's ataxia, and it is safe to predict that the treatment space will significantly impact the Friedreich's ataxia market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the Friedreich's ataxia market in the 7MM. However, several factors may impede the growth of the Friedreich's ataxia market. Progressive neurodegeneration in Friedreich's ataxia causes irreversible damage to the spinal cord, peripheral nerves, and cerebellum, leading to cumulative and disabling impairments; however, diagnostic delays due to reliance on clinical evaluation and genetic testing, especially in atypical or late-onset cases, combined with challenges in clinical trial design stemming from small patient populations, disease heterogeneity, and limited validated endpoints, as well as financial barriers such as high R&D costs and reimbursement hurdles, collectively complicate drug development and restrict patient access to emerging therapies. Moreover, Friedreich's ataxia treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the Friedreich's ataxia market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Friedreich's ataxia market growth. Friedreich's Ataxia Treatment Market Current treatments for Friedreich's ataxia primarily focus on alleviating specific symptoms rather than addressing the root cause of the disease. Despite advancements, symptom management continues to be the standard approach to care. While there is no cure, several strategies can help improve the quality of life for those affected. The most serious complication, heart disease, can often be managed with medications commonly used in the general population, such as ACE inhibitors, beta-blockers, and diuretics, which help reduce cardiac strain. In cases of irregular heart rhythms, pacemakers or drugs may help stabilize heart function. Diabetes, another potential symptom, can be treated with insulin. Surgical interventions are occasionally required to address foot deformities or scoliosis; though significant, these procedures are generally effective. Physical therapy plays a crucial role in maintaining muscle flexibility and mobility, while speech therapy can assist in improving communication and swallowing abilities by retraining facial and tongue muscles. Emerging research targeting the root cause of Friedreich's ataxia focuses on frataxin, a protein involved in iron regulation and oxidative stress. Gene therapy approaches have shown potential, especially gene replacement strategies, although issues like targeted delivery, genotoxicity, and regulation of gene expression remain unresolved. Alternative approaches such as protein replacement using frataxin precursors or TAT-frataxin fusion proteins are also under investigation. SKYCLARYS (omaveloxolone) is approved for treating Friedreich's ataxia in patients aged 16 and older, though its safety and effectiveness in younger individuals are still unknown. Omaveloxolone (RTA-408) is a semisynthetic oleanane triterpenoid with anti-inflammatory and antioxidant effects. It activates the Nrf2 transcription factor, which is crucial for controlling oxidative stress, a function impaired in Friedreich's ataxia due to mitochondrial dysfunction. Enhancing Nrf2 activity through omaveloxolone may thus offer therapeutic benefits. In September 2023, Biogen finalized its acquisition of Reata Pharmaceuticals, the company behind omaveloxolone. To know more about Friedreich's ataxia treatment options, visit @ Friedreich's Ataxia Medication Friedreich's Ataxia Pipeline Therapies and Key Companies Some of the drugs in the pipeline include RT001 (Retrotope), Vatiquinone (PTC Therapeutics), Leriglitazone (Minoryx Therapeutics), Omaveloxolone (Biogen), LX2006 (Lexeo Therapeutics), CTI-1601 (Larimar Therapeutics, Inc.), SGT-212 (Solid Biosciences), and others. Vatiquinone, developed by PTC Therapeutics, is an experimental small molecule that targets 15-Lipoxygenase, an enzyme critically involved in regulating oxidative stress and inflammatory responses linked to neurological disorders. In March 2014, the U.S. FDA granted it Fast Track designation for treating Friedreich's ataxia, and it also holds Orphan Drug designation for the same condition. Currently, vatiquinone is in Phase III clinical trials for Friedreich's ataxia. In February 2025, the FDA accepted its New Drug Application (NDA) for the treatment of both pediatric and adult patients with Friedreich's ataxia, awarding it Priority Review with a PDUFA target action date of August 19, 2025. RT001, developed by Retrotope, is a deuterated ethyl linoleate that inhibits lipid peroxidation, aiming to mitigate cellular damage and improve mitochondrial function. It received Fast Track status from the FDA in February 2021, and had earlier been granted Orphan Drug status in 2016 for Friedreich's ataxia. A Phase II/III trial evaluating its safety and efficacy in Friedreich's ataxia was completed in August 2021. Leriglitazone, a product of Minoryx Therapeutics, is an orally active, selective PPARγ agonist with a promising profile for central nervous system (CNS) disorders. It was designated an Orphan Drug by both the FDA and EMA in 2019 for Friedreich's ataxia. Clinical evaluation in Phase II trials showed beneficial outcomes in patients with the condition. The anticipated launch of these emerging therapies are poised to transform the Friedreich's ataxia market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Friedreich's ataxia market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. Discover more about Friedreich's ataxia treatment pipeline @ Friedreich's Ataxia Clinical Trials Recent Developments in the Friedreich's Ataxia Market In April 2025, Lexeo Therapeutics, Inc. reported encouraging interim results from all dose groups of LX2006 in the treatment of Friedreich ataxia cardiomyopathy. Data from both the Lexeo-sponsored SUNRISE-FA Phase 1/2 trial (NCT05445323) and the investigator-initiated Phase 1A trial at Weill Cornell Medicine (NCT05302271) showed that LX2006 led to meaningful improvements in cardiac biomarkers and functional outcomes. Additionally, all participants who underwent cardiac biopsies demonstrated increased frataxin protein expression. In February 2025, the FDA accepted the New Drug Application (NDA) for vatiquinone, intended for the treatment of both children and adults with Friedreich's ataxia. The application has been granted Priority Review. In January 2025, Solid Biosciences Inc. announced that the FDA has granted Fast Track designation to SGT-212, its AAV-based gene therapy candidate for treating Friedreich's ataxia. Friedreich's Ataxia Overview Friedreich's ataxia is a rare genetic disorder that results in progressive damage to the nervous system and problems with movement. It typically starts during childhood and gradually leads to worsening muscle coordination. The condition is caused by a mutation in the FXN gene, which provides instructions for producing a protein called frataxin. People who inherit two faulty copies of this gene, one from each parent, develop the disease. In atypical cases, Friedreich's ataxia is classified into two subtypes: late-onset FRDA (LOFA), which begins between the ages of 26 and 39, and very late-onset FRDA (VLOFA), which starts after age 40. Symptoms generally appear between the ages of 5 and 15, with the earliest signs often including difficulty walking, poor balance, and slurred or slow speech. Diagnosing Friedreich's ataxia involves a detailed clinical evaluation, including a comprehensive physical examination to identify issues such as balance problems, loss of joint position sense, absent reflexes, and other neurological signs. In addition, genetic testing now offers a definitive method for confirming the diagnosis. Friedreich's Ataxia Epidemiology Segmentation The Friedreich's ataxia epidemiology section provides insights into the historical and current Friedreich's ataxia patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The Friedreich's ataxia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Diagnosed Prevalent Cases of Friedreich's Ataxia Onset Type Diagnosed Prevalent Cases of Friedreich's Ataxia Total Treated Cases of Friedreich's Ataxia Scope of the Friedreich's Ataxia Market Report Therapeutic Assessment: Friedreich's Ataxia current marketed and emerging therapies Friedreich's Ataxia Market Dynamics: Key Market Forecast Assumptions of Emerging Friedreich's Ataxia Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Friedreich's Ataxia Market Access and Reimbursement Download the report to understand which factors are driving Friedreich's ataxia market trends @ Friedreich's Ataxia Treatment Market Table of Contents Related Reports Friedreich's Ataxia Epidemiology Forecast Friedreich's Ataxia Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Friedreich's ataxia epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Friedreich's Ataxia Pipeline Friedreich's Ataxia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Friedreich's ataxia companies, including PTC Therapeutics, Retrotope, Reata Pharmaceuticals, Minoryx Therapeutics, Larimar Therapeutics, LEXEO Therapeutics, Exicure, StrideBio, Voyager Therapeutics, Lacerta Therapeutics, among others. Ataxia Market Ataxia Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key ataxia companies, including AstraZeneca, Merck Sharp & Dohme LLC, Bayer, Roche, among others. Ataxia Pipeline Ataxia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ataxia companies, including Retrotope, Reata Pharmaceuticals, PTC Therapeutics, Metro International Biotech, LLC, Design Therapeutics, Larimar Therapeutics, Minoryx Therapeutics, EryDel, Biogen, Matrix Biomed, IntraBio, Biohaven Pharmaceuticals, Inc., Stealth BioTherapeutics Inc., Acasti Pharma, Seelos Therapeutics, Kissei Pharmaceutical, Vico Therapeutics, Q-State Biosciences, Locanabio, Lexeo Therapeutics, Voyager Therapeutics, CRISPR Therapeutics, Capsida Biotherapeutics, AavantiBio, StrideBio, Wave Life Sciences, REPROCELL, SHIONOGI & Co., CORESTEM, Blade Therapeutics, Exicure, Lacerta Therapeutics, Healx Ltd., Uniqure, Ionis Pharmaceuticals, Jupiter Neurosciences, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Orphan DrugFast TrackDrug ApprovalPhase 2

100 Deals associated with NNC0268-0965

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Diabetes Mellitus, Type 2	Phase 1	Germany	Novo Nordisk A/S	29 Oct 2019
Diabetes Mellitus, Type 1	Phase 1	Germany	Novo Nordisk A/S	05 Jun 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


WSC2023	Not Applicable	Sleep Apnea Syndromes	-	Diabetic Patients on Insulin Therapy	viyxymclgo(selyskjpdu) = wifrhovskh mbnnrdzfvi (qtztovnsot, 0.02) View more	-	23 Oct 2023
STOP study (EASD2023)	Not Applicable	Diabetes Mellitus, Type 2	93	IDegLira	fvmfiguoih(pfvowdmfja) = cesybwigqd fdhhcjwbqk (pfbevvjkpw ) View more	Positive	05 Oct 2023
EASD2023	Not Applicable	-	-	Open-source automated insulin delivery (osAID) system	zpyqpzocod(okjfpnpnwf) = wqgmuxfekm xhlllgvfxz (qncggmmalt ) View more	Positive	05 Oct 2023
				insulin (Medtronic 780G with SmartGuard)	zpyqpzocod(okjfpnpnwf) = knphcihabh xhlllgvfxz (qncggmmalt ) View more
EASD2023	Not Applicable	Diabetes Mellitus, Type 1 \| Diabetic Retinopathy \| Retinal Diseases	652	automated insulin delivery systems (Multiple Daily Injections (MDI))	rfrigknlzz(rxfogqqyka) = Significantly lower rates of dyslipidemia in patients with AID were seen weqwkdlnjq (tevkgkbuaq ) View more	Positive	05 Oct 2023
				automated insulin delivery systems (Insulin Pump (IP) without AID)
EASD2023	Not Applicable	Diabetes Mellitus, Type 2	514	insulin (Descending mode)	cstfpkyrew(wzlmmzxhjr) = zutyyduxnd wpujfyfsdq (xwsdgnvwdd, (1.0 - 6.0)) View more	Positive	05 Oct 2023
				insulin (Ascending mode)	cstfpkyrew(wzlmmzxhjr) = tltgsyphib wpujfyfsdq (xwsdgnvwdd, (0 - 4.0)) View more
INSENODIAB (ESPE2023)	Not Applicable	Diabetes Mellitus, Type 1	-	Insulin	kmyfwscujj(hxiveksybg) = jtyojbtlvr gdijxosrmy (yhpagdbffq, 0.5)	-	21 Sep 2023
				rINSENODIAB cohort	kmyfwscujj(hxiveksybg) = yrpcykjfya gdijxosrmy (yhpagdbffq, 6.9)
ESPE2023	Not Applicable	Familial chylomicronaemia syndrome LPL deficiency	4	Insulin	tgjlwywjsh(mgcibebrsi) = kdthdjgtsv ffyabcgzzu (sezqwkpvdt )	-	21 Sep 2023
ADA2023	Not Applicable	Diabetes Mellitus, Type 2	96	Basal insulin only	bjzgapqvyl(lxeyklkyzq) = tgaljoxrhm vyysksubpu (jkfxtzcoya ) View more	-	23 Jun 2023
				Multiple daily injections (MDI)	bjzgapqvyl(lxeyklkyzq) = errnqjylac vyysksubpu (jkfxtzcoya ) View more
ADA2023	Not Applicable	Pregnancy	-	Concentrated insulin in continuous subcutaneous insulin infusion (CSII)	nxbynfrkou(ynpoycqbcr) = hlydzbmumg uqwzyyxwqu (ptuiixzewh ) View more	-	20 Jun 2023
ADA2023	Not Applicable	-	-	Insulin + Glucagon	hdyutwhhtl(kefectwzyb) = ghqppovbte xtylmvbhan (qdjqimqgzk )	-	20 Jun 2023
				Insulin	hdyutwhhtl(kefectwzyb) = xxnarhdkek xtylmvbhan (qdjqimqgzk )

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Biosimilar

Competitive landscape of biosimilars in different countries/locations. Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis