Long-term Follow-up Safety and Efficacy Study in Participants With Duchenne Muscular Dystrophy Who Have Received Fordadistrogene Movaparvovec in a Preceding Clinical Study

The purpose of this study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies. We will follow participants' experience in this study for 10 years after the end of their previous study. Participants will have 1 annual onsite visit and a few annual remote visits. The exact number of remote visits will be decided by their study doctor.

Start Date13 Mar 2023

Sponsor / Collaborator

Pfizer Inc.

NCT05429372

/ Active, not recruitingPhase 2

A PHASE 2, MULTICENTER, SINGLE-ARM STUDY TO EVALUATE THE SAFETY AND DYSTROPHIN EXPRESSION AFTER FORDADISTROGENE MOVAPARVOVEC (PF-06939926) ADMINISTRATION IN MALE PARTICIPANTS WITH EARLY STAGE DUCHENNE MUSCULAR DYSTROPHY

The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study

Start Date08 Aug 2022

Sponsor / Collaborator

Pfizer Inc.

NCT04281485

/ Active, not recruitingPhase 3

A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF 06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.

Start Date05 Nov 2020

Sponsor / Collaborator

Pfizer Inc.

100 Clinical Results associated with Fordadistrogene movaparvovec

100 Translational Medicine associated with Fordadistrogene movaparvovec

100 Patents (Medical) associated with Fordadistrogene movaparvovec

Literatures (Medical) associated with Fordadistrogene movaparvovec

01 Jun 2025·MOLECULAR THERAPY

Complement activation in a phase Ib study of fordadistrogene movaparvovec for Duchenne muscular dystrophy

Article

Author: Smith, Edward C ; Ryan, Kelly ; Ravindra, Kodihalli C ; Casinghino, Sandra ; Sirivelu, Madhu P ; Schulz, Martin ; Delnomdedieu, Marielle ; Binks, Michael ; Licht, Christoph ; Shi, Heliang ; Shen, Qi ; Byrne, Barry J ; Vaidya, Vishal S ; Shieh, Perry B ; McDonnell, Tara ; Whiteley, Laurence ; Levy, Daniel I ; Butterfield, Russell J

Thrombotic microangiopathy (TMA) has been reported as an uncommon but severe adverse effect of recombinant adeno-associated virus (rAAV)-based gene therapy. Here, we describe in detail the occurrences of clinically evident TMA following infusion of the rAAV9-based therapy fordadistrogene movaparvovec (1 or 3 × 10¹⁴ vg/kg) in 22 participants with Duchenne muscular dystrophy (DMD). Three participants experienced clinical evidence of TMA. Platelet levels rapidly decreased 7-10 days post infusion, together with C3 and C4 depletion and elevated C5b-9 after ∼7 days. Peak vector blood concentration was observed for 4-7 days, and type 1 interferon cytokine levels increased within 2-7 days. Each affected participant was hospitalized and received supportive care and anti-complement therapy. Anti-AAV9 immunoglobulin M (IgM) and IgG were detected within days post infusion, and participants with TMA demonstrated a particularly rapid rise of neutralizing antibodies and total antibody to AAV9 in the first 1-2 weeks post infusion. Limited early time points from other participants were not assessed for complement activation. With appropriate monitoring of early time points following AAV exposure, TMA can be successfully managed. However, these early events should be considered related to the benefit-risk profile when using rAAV-based gene therapy for the treatment of DMD. ClinicalTrials.gov identifier: NCT03362502.

01 Jun 2025·MOLECULAR THERAPY

Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchenne muscular dystrophy: Initial observations from a phase 1b trial

Article

Author: Sherlock, Sarah P ; Ryan, Kelly A ; McIntosh, Avery ; Shieh, Perry B ; Binks, Michael ; Lal, Ashwin K ; Butterfield, Russell J ; McDonnell, Tara G ; Delnomdedieu, Marielle ; Levy, Daniel I ; Smith, Edward C

Fordadistrogene movaparvovec (FM; PF-06939926) is a recombinant adeno-associated virus serotype-9 gene-replacement construct containing a mini-dystrophin transgene in development for Duchenne muscular dystrophy (DMD). We present findings of cardiac safety assessments in participants with DMD during a 1-year follow-up from an ongoing phase 1b multicenter, single-arm, open-label trial of low- and high-dose FM. Cardiac troponin-I (cTn-I) levels and cardiac magnetic resonance imaging measures were obtained from 19 ambulatory participants (n = 3, low dose; n = 16, high dose; median age 8.8 years) and 3 non-ambulatory participants (high dose; median age 15.1 years). Six ambulatory and 3 non-ambulatory participants had cTn-I levels above the upper limit of normal at baseline. Of these, 1 ambulatory participant and 2 non-ambulatory participants had cTn-I levels >3× the baseline level during the first year following infusion. At 1 year post-infusion, mean (±SD) changes from baseline in left ventricular ejection fraction (LVEF) were -0.9% ± 4.0% in ambulatory participants and -3.1% ± 1.8% in non-ambulatory participants. One 16-year-old non-ambulatory participant with DMD experienced fatal cardiogenic shock 6 days after a high dose of FM. With the exception of participants with DMD with advanced cardiac fibrosis, assessments of cTn-I, LVEF by cardiac magnetic resonance imaging and progression of late gadolinium enhancement suggest low cardiac toxicity from FM in ambulatory participants with DMD in this study. ClinicalTrials.gov identifier: NCT03362502.

01 Sep 2023·Molecular therapy. Methods & clinical development

Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy

Article

Author: Xiao, Xiao ; Grieger, Josh ; McIntyre, Maritza ; Adjali, Oumeya ; Remy, Séverine ; Le Guiner, Caroline ; Samulski, R Jude ; Larcher, Thibaut ; Huchet, Corinne ; Owens, Jane ; Lafoux, Aude ; Li, Juan ; Anegon, Ignacio ; Farrokhi, Vahid ; Toumaniantz, Gilles ; Neubert, Hendrik ; Moullier, Philippe

Duchenne muscular dystrophy (DMD) is an X-linked disease caused by loss-of-function mutations in the dystrophin gene and is characterized by muscle wasting and early mortality. Adeno-associated virus-mediated gene therapy is being investigated as a treatment for DMD. In the nonclinical study documented here, we determined the effective dose of fordadistrogene movaparvovec, a clinical candidate adeno-associated virus serotype 9 vector carrying a human mini-dystrophin transgene, after single intravenous injection in a dystrophin-deficient (DMD^mdx) rat model of DMD. Overall, we found that transduction efficiency, number of muscle fibers expressing the human mini-dystrophin polypeptide, improvement of the skeletal and cardiac muscle tissue architecture, correction of muscle strength and fatigability, and improvement of diastolic and systolic cardiac function were directly correlated with the amount of vector administered. The effective dose was then tested in older DMD^mdx rats with a more dystrophic phenotype similar to the pathology observed in older patients with DMD. Except for a less complete rescue of muscle function in the oldest cohort, fordadistrogene movaparvovec was also found to be therapeutically effective in older DMD^mdx rats, suggesting that this product may be appropriate for evaluation in patients with DMD at all stages of disease.

News (Medical) associated with Fordadistrogene movaparvovec

24 Jun 2025

·www.einpresswire.com

Duchenne Muscular Dystrophy (DMD) Gene Therapy: Competitive Intelligence for Pharma Leadership | DataM Intelligence

The article analyzes competition in DMD gene therapies, spotlighting FDA approvals, high costs, & strategic positioning of treatments like Givinostat & Elevidys The race to dominate Duchenne therapy is fierce. Givinostat’s oral HDAC edge meets Elevidys’ micro‑dystrophin promise-but sky‑high prices and safety signals may shape future winners.” — DataM Intelligence AUSTIN, TX, UNITED STATES, June 24, 2025 / EINPresswire.com / -- 1. Disease Overview & Molecular Pathogenesis Duchenne Muscular Dystrophy (DMD) is an X-linked recessive neuromuscular disorder resulting from mutations in the dystrophin gene, which encodes a structural protein critical for muscle fiber stability. Mutations lead to absent or non-functional dystrophin, causing muscle cell fragility, progressive myofiber degeneration, and eventual replacement with fibrofatty tissue. Clinical onset typically occurs by age five, with progressive weakness, loss of ambulation in early adolescence, and death in the late teens to early 20s due to respiratory or cardiac failure. 2. Epidemiology & Market Forecasts DMD affects approximately 1 in 3,500 to 5,000 live male births globally. The therapeutics market, valued at around USD 2.1–2.3 billion in 2023–2024, is projected to grow rapidly, potentially reaching USD 7.4–9.9 billion by 2030–2034. Growth is driven by the expansion of exon-skipping therapies, the entry of gene therapy platforms, increasing diagnosis through newborn screening, and the development of oral and mutation-agnostic treatment modalities. Compound annual growth rates (CAGR) are expected to range between 14% and 40%, depending on regulatory approvals and therapeutic uptake. Download CI Sample Report: https://www.datamintelligence.com/strategic-insights/ci/duchenne-muscular-dystrophy-gene-therapy-competitive-edge-insights 3. Current Treatment Paradigms Exon-Skipping Therapies Antisense oligonucleotides (ASOs) are designed to skip specific exons during pre-mRNA splicing to restore the dystrophin reading frame. Approved agents include: - Eteplirsen for exon 51 - Golodirsen and Viltolarsen for exon 53 - Casimersen for exon 45 These therapies target mutation-specific subpopulations and require weekly IV infusions. Functional benefit is often modest, and long-term outcomes remain under evaluation. Steroidal and Non-Steroidal Orals - Vamorolone, a dissociative corticosteroid, offers anti-inflammatory activity with a reduced side effect profile. It is approved for children aged 6 and older, regardless of mutation type. - Givinostat, a histone deacetylase inhibitor (HDACi), represents the first approved non-steroidal agent with disease-modifying properties, showing promise in reducing muscle fibrosis and preserving function. Gene Therapy - Elevidys (delandistrogene moxeparvovec) is the first FDA-approved AAV-based gene therapy for DMD. It delivers a truncated micro-dystrophin gene via a single IV dose, aiming for durable expression. Initially approved for ambulatory children aged 4–5, Elevidys is now expanding to non-ambulatory populations. However, concerns over liver toxicity, patient deaths in trials, and uncertain durability present critical hurdles. 4. Emerging Pipeline Modalities - Next-Gen AAV Gene Therapies: Several candidates, including RGX-202, SGT-003, and GNT0004, are being developed to enhance transduction efficiency, expand age eligibility, and reduce immunogenicity. - CRISPR/Cas Gene Editing: Preclinical programs aim for single-dose, mutation-independent correction of the dystrophin gene, with potential for a curative approach. - Cell-Based Therapies: Agents like CAP-1002, derived from cardiosphere-derived cells, are being evaluated for their anti-inflammatory and regenerative effects. - Adjunctive Agents: Anti-fibrotic and muscle-stabilizing molecules are being developed to complement dystrophin-restoring treatments, particularly in non-ambulatory stages. 5. Market Access & Commercialization Challenges - Cost Barriers: AAV gene therapies are priced in the multi-million-dollar range. For example, Elevidys costs approximately USD 3.2 million per patient, requiring innovative value-based pricing models. - Regulatory Heterogeneity: While accelerated approvals enable early access, global variation in reimbursement policies complicates commercialization, especially in Europe and Asia-Pacific. - Patient Access Gaps: Most therapies target ambulatory children under 5 years old, leaving older or non-ambulatory patients underserved-a significant market expansion opportunity. - Safety and Durability: AAV-based therapies face scrutiny over immune responses, hepatotoxicity, and uncertain long-term benefit, necessitating rigorous post-marketing surveillance and real-world data collection. Book Free CI Consultation Call: https://www.datamintelligence.com/strategic-insights/ci/duchenne-muscular-dystrophy-gene-therapy-competitive-edge-insights 6. Competitive Landscape: Key Companies and Strategies Sarepta Therapeutics - The current market leader, Sarepta offers the most extensive exon-skipping portfolio and the first approved gene therapy (Elevidys). The company is strategically expanding its label to non-ambulatory populations and building a robust global infrastructure for commercial distribution and real-world data capture. Pfizer - Developing an AAV-based gene therapy (PF-06939926), Pfizer is conducting Phase III trials with a broader patient population than Sarepta’s initial cohort. Pfizer leverages its large-scale manufacturing and global commercial footprint to compete in delivery and scalability. Regenxbio - Its RGX-202 program features proprietary vector technology targeting skeletal muscle for enhanced gene transduction. The program is advancing through Phase II/III studies with a focus on increased dystrophin production and improved durability. Solid Biosciences - This company’s SGT-003 therapy uses a muscle-specific promoter to improve gene expression specificity and safety. Its differentiated vector design and targeted delivery approach aim to overcome limitations of first-generation AAV therapies. NS Pharma (Nippon Shinyaku) - NS Pharma has commercialized viltolarsen (exon 53 skipping) and is pushing for regulatory approval in Europe. The company is focusing on deepening its foothold in exon-skipping with region-specific partnerships and accelerated review designations. Santhera Pharmaceuticals / Catalyst Pharmaceuticals - Vamorolone, their non-steroidal anti-inflammatory steroid alternative, addresses all mutation types and offers an improved side effect profile over corticosteroids, positioning it for widespread uptake in early and maintenance stages of DMD. Italfarmaco - Developer of givinostat, Italfarmaco is targeting muscle fibrosis reduction and functional stabilization. Its HDAC inhibition mechanism offers a unique value proposition as an adjunct to exon-skipping or gene therapy. Emerging Genome Editing Startups - Multiple biotech firms are pursuing CRISPR-based and epigenetic editing strategies to develop curative therapies. These programs, still in preclinical stages, aim to correct the underlying mutation at the genomic level, with potential long-term disease reversal. Ask For Customized CI Service As per Your Business Requirement: https://www.datamintelligence.com/strategic-insights/ci/duchenne-muscular-dystrophy-gene-therapy-competitive-edge-insights 7. Strategic Imperatives for Pharma Stakeholders Pipeline Optimization - Pharma firms must prioritize mutation-independent platforms (e.g., gene editing, full-length dystrophin delivery) and invest in strategies that extend eligibility to older, non-ambulatory patients. Developing combination regimens that include anti-fibrotic agents or oral steroids could further differentiate portfolios. Clinical Trial Innovation - Designing biomarker-rich trials that quantify dystrophin levels, muscle strength, and MRI-detected tissue changes will be essential. Adaptive trials and global multi-site platforms can expedite enrollment and regulatory alignment. Market Access Readiness - Early engagement with payers to shape outcomes-based pricing, annuity payment models, and evidence-based value frameworks will be critical for therapies with high upfront costs. Demonstrating reductions in lifetime disease burden, hospitalizations, and caregiver strain will strengthen economic positioning. Safety and Immunogenicity Management - Companies must invest in pre-treatment screening protocols, liver monitoring guidelines, and immunosuppressive strategies to mitigate the risks associated with viral vector delivery. Transparent communication with regulators and stakeholders regarding adverse events will build long-term trust. Integrated Care Strategy - Support services including home infusion options, digital adherence platforms, and tele-neurology networks will be essential for long-term treatment success and patient engagement. Partnerships with neuromuscular centers can serve as launchpads for patient identification and retention. Diagnostics and Early Intervention - Expansion of newborn screening and carrier detection programs will increase early diagnosis and support early therapeutic intervention-vital for maximizing the efficacy of gene therapies and exon-skipping agents. Global Expansion Strategy - Building tiered pricing frameworks, regional licensing partnerships, and local manufacturing strategies will be crucial for entering emerging markets. Pharma players must prepare to balance innovation with affordability to secure broad international adoption. Read Related CI Reports: 1. Alpha 1 Antitrypsin Deficiency | Competitive Intelligence 2. Chronic Spontaneous Urticaria | Competitive Intelligence Sai Kumar DataM Intelligence 4market Research LLP +1 877-441-4866 sai.k@datamintelligence.com Visit us on social media: LinkedIn X Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Phase 3Gene TherapyDrug ApprovalOligonucleotide

03 Apr 2025

·www.biospace.com

Sarepta’s Duchenne Gene Therapy Placed on Hold in Europe Pending Death Investigation

Courtesy of Sarepta Therapeutics The European Medicines Agency asked for three clinical trials to be placed on hold until the exact cause of death of a US teenager, who suffered acute liver failure after receiving Elevidys, can be precisely determined. The European Medicines Agency has requested that Sarepta Therapeutics and partner Roche pause a trio of clinical trials of their Duchenne muscular dystrophy gene therapy Elevidys in response to a patient death that occurred outside the scope of the studies. In a letter addressed to the World Duchenne Organization and dated March 31, Roche shared that it was pausing Phase I , Phase II and Phase III trials all studying the gene therapy for treating Duchenne muscular dystrophy (DMD). The European Medicines Agency (EMA) asked for the trials to be paused until the exact cause of death of a U.S. teenager , who had suffered acute liver failure (ALF) after taking Elevidys, can be precisely determined. Sarepta announced last month at the time of the patient’s death that it would update Elevidys’ label to reflect the safety signal. Analysts at BMO were nonplussed by the trial pause. “We view the requested clinical hold as an administrative step, pending [data monitoring committee] meeting, that can be resolved within ~6mo given Elevidys benefit/risk in the context of DMD being a universally fatal disease ,” the analysts wrote in a note late Wednesday (emphasis theirs). Other analysts were more mixed. “On one hand, the risk of fatal ALF is rare (0.125%) and our experts affirm the news is unsurprising to them. However, we’ve felt the death should not be fully dismissed unless [Sarepta management] can provide color on some outstanding questions,” Jefferies analysts wrote Wednesday. The connection between biopharmas working on DMD and the patient community is especially tight knit . Following the patient’s death, Sarepta sent a letter directly to the DMD patient community, pledging transparency and sharing data as the company investigates the death. The sentiment mirrors those reflected in Roche’s letter earlier this week to the DMD community. The company signed off saying, “We truly appreciate your leadership and the opportunity to work together with you in these efforts.” The patient death after taking Elevidys is one of a series of deaths from DMD gene therapies. Two patients died, one in 2021 and another in 2024 , while taking Pfizer’s investigational gene therapy fordadistrogene movaparvovec. That program was later scrapped. In 2022, a CRISPR-based Duchenne therapy led to another patient death, potentially caused by the viral vector used.

Gene TherapyPhase 3Phase 2

18 Mar 2025

·www.biospace.com

Sarepta Shares Crash 22% After Patient on DMD Gene Therapy Elevidys Dies

Sarepta Therapeutics After a patient taking the Duchenne muscular dystrophy gene therapy Elevydis died of liver injury, Sarepta will update the label to reflect the safety signal. A patient taking Sarepta Therapeutics ’ approved Duchenne muscular dystrophy gene therapy Elevidys has died , the biotech announced Tuesday. The death was linked to acute liver failure, which Sarepta maintains is not a new safety signal. Elevidys and other gene therapies that use adeno-associated virus (AAV) vectors are known to have acute liver injury as a side effect and that risk is highlighted as an adverse event in Elevidys’ prescribing information . Sarepta also insists that the gene therapy’s benefit-risk profile “remains positive.” Sarepta has reported the death to the relevant health authorities and will update Elevidys’ label to “appropriately represent this event,” the biotech noted in its Tuesday release. The company’s stock plunged around 22% as the markets opened on Tuesday to $78.54, down from $101.35 at the previous market close. The death “represents a severity of acute liver injury not previously reported for Elevidys,” Sarepta noted. The patient had also tested positive for cytomegalovirus (CMV) infection, which can damage the liver and is “a possible contributing factor” to the death, according to the announcement. Sarepta expressed “profound” sadness over the death. BMO Capital Markets said in a Tuesday note that despite the death, “we think Elevidys’ benefit still outweighs the risk,” pointing to the patient’s CMV infection as a mitigating actor. BMO also highlighted two patients who died after taking Novartis’ spinal musclar atrophy gene therapy drug Zolgensma in 2022. That drug today generates north of $1 billion annually. In another note on Tuesday, analysts at Jefferies said they expect the patient death will “pressure” Sarepta’s stock as “uncertainty around [Elevidys’] safety will loom—unless [Sarepta] deems the death not to be drug-related.” Nevertheless, Jefferies points to a potential bright spot for Sarepta, noting that liver injury typically occurs within the first 90 days of Elevidys treatment. “Presumably, the patient was dosed with Elevidys recently,” the analysts continued, adding “mitigating these types of events could be manageable with very careful monitoring.” Jefferies also pointed out that “the unmet need in DMD [Duchenne muscular dystrophy] remains high,” which could be a “bull case” for Sarepta. Elevidys isn’t the first Duchenne therapy to be linked to a fatal outcome. In 2021, Pfizer was forced to suspend a Phase Ib trial for its investigational gene therapy PF-06939926—which would later get renamed as fordadistrogene movaparvovec—following the death of a non-ambulatory patient. Three years later, in March 2024, the same therapy resulted in another patient death in the Phase II DAYLIGHT trial. A month later, fordadistrogene movaparvovec failed to significantly improve motor function versus placebo in the Phase III CIFFREO trial in Duchenne. Pfizer ultimately axed the gene therapy. Meanwhile, in November 2022, an FDA-sanctioned trial from the non-profit biotech Cure Rare Disease for a CRISPR-based Duchenne therapy also led to the death of the only enrolled patient. A study in September 2023 pointed to the AAV vector as the potential cause of the mortality, which could have triggered an immune reaction in the patient.

Gene TherapyPhase 2Phase 3

100 Deals associated with Fordadistrogene movaparvovec

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	Phase 3	United States	Pfizer Inc.	05 Nov 2020
Muscular Dystrophy, Duchenne	Phase 3	Japan	Pfizer Inc.	05 Nov 2020
Muscular Dystrophy, Duchenne	Phase 3	Australia	Pfizer Inc.	05 Nov 2020
Muscular Dystrophy, Duchenne	Phase 3	Belgium	Pfizer Inc.	05 Nov 2020
Muscular Dystrophy, Duchenne	Phase 3	Canada	Pfizer Inc.	05 Nov 2020
Muscular Dystrophy, Duchenne	Phase 3	France	Pfizer Inc.	05 Nov 2020
Muscular Dystrophy, Duchenne	Phase 3	Germany	Pfizer Inc.	05 Nov 2020
Muscular Dystrophy, Duchenne	Phase 3	Israel	Pfizer Inc.	05 Nov 2020
Muscular Dystrophy, Duchenne	Phase 3	Italy	Pfizer Inc.	05 Nov 2020
Muscular Dystrophy, Duchenne	Phase 3	Russia	Pfizer Inc.	05 Nov 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04281485 (CIFFREO, Biospace) Manual	Phase 3	Muscular Dystrophy, Duchenne	-	fordadistrogene movaparvovec	cejwavprsp(clmmkhtxne) = the gene therapy candidate was unable to significantly improve motor function in boys aged four to seven years, compared to placebo ryyucohfxq (ozyodepiod ) View more	Negative	13 Jun 2024
MDA2024	Phase 1	Muscular Dystrophy, Duchenne	16	Fordadistrogene movaparvovec (FM)	kdhskirfsl(bkvshfwgyt) = hkbyyhbsoo vngphyxinu (cxojftzgaz, 17.0)	Positive	03 Mar 2024
				Fordadistrogene movaparvovec (External control group)	kdhskirfsl(bkvshfwgyt) = tuyjpdpgxj vngphyxinu (cxojftzgaz, 18.8)
MDA2024	Not Applicable	Muscular Dystrophy, Duchenne	-	Fordadistrogene movaparvovec low-dose	rpugdsresw(klmxyhnbtq) = bcmrukaesy fzawunkmby (zvbsafnzfq, 1.8)	-	03 Mar 2024
				Fordadistrogene movaparvovec high-dose	rpugdsresw(klmxyhnbtq) = auvinwpkem fzawunkmby (zvbsafnzfq, 2.1)
ESGCT2023	Not Applicable	Muscular Dystrophy, Duchenne	-	Fordadistrogene movaparvovec (FM)	iftcyfiwkv(ujufknomwg) = dwnovgokef xmnhmgbxfd (jrjezrqfmd ) View more	-	24 Oct 2023
				Domagrozumab	iftcyfiwkv(ujufknomwg) = zcrzzaupbr xmnhmgbxfd (jrjezrqfmd ) View more
NCT03362502 (MDA2022)	Phase 1	Muscular Dystrophy, Duchenne	-	Fordadistrogene movaparvovec (low-dose)	gvhxuwiezo(pjqtzxgvxy) = 3 treatment-related serious adverse events occurred (dehydration, acute kidney injury, thrombocytopenia); all resolved within 15 days vcbdyoegnj (jnapsgxeny ) View more	-	13 Mar 2022

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