Chondrodysplasia Punctata, Rhizomelic

MOOSE JAW, SK, July 23, 2024 /PRNewswire/ -- World renowned neuroscientist, inventor, and entrepreneur, Dr. Dayan Goodenowe, is bringing his advanced restorative health technology to his hometown of Moose Jaw. Dr. Goodenowe is Launching a bold multimillion dollar, multi-year initiative called the Moose Jaw Vitality Project. The project will offer free community programs for challenging health conditions from autism to Alzheimer's with the objective of making Moose Jaw the healthiest city in North America. The first program to be offered is the Restorative Health in Autism Community Program. All eligible children and young adults with autism in Moose Jaw from age 2 to 30 are invited to enroll. Families will be provided with advanced education in autism, targeted brain nutritive supplements, monitoring, and support - all completely free of charge. A core component of restoring brain health in autism is resolving inflammation and restoring myelination. Plasmalogens are natural lipid molecules made by the brain. Plasmalogen precursors are natural nutritive ingredients found mostly in human breast milk. Dr. Goodenowe was the first scientist to invent, patent, manufacture, and clinically validate plasmalogen precursors. Today, over 2500 doctors and practitioners around the world use Dr. Goodenowe's plasmalogens in the routine care of their patients. Over a thousand parents of autistic children have experienced improvements in their child's mental health and well-being with plasmalogen precursors. It is now time to come together as a community with science, compassion, and love to show the world how to restore function and quality of life in children and adults with autism that will last a lifetime. Plasmalogens are critical building blocks essential for normal brain development in children, especially the protective wrapping on the brain's wires (called axons). This protective wrapping is called myelin (or white matter). Numerous rare childhood diseases in children either directly (such as Rhizomelic Chondrodysplasia Punctata (RCDP)) or indirectly (such as leukodystrophies and mitochondrial defect diseases) disrupt normal brain myelin development resulting in symptoms often indistinguishable from autism. In addition, there are numerous other ways that normal brain development can be disrupted (prematurity, head injury, infections, and exposure to toxins) resulting in autism or autism-like symptoms. The community of Moose Jaw is invited to a special two-day event filled with fun, food, and groundbreaking information on Autism and Restorative Neurology. The two-day event kicks off with an Open House of the Dr. Goodenowe Restorative Health Center (1350 Lakeview Road) on July 30th, the grand opening of the Dr. Goodenowe Community Health Center (Town and Country Mall) on July 31 and culminating with a special evening event (5PM at Connor Park) on July 31st where you will get to meet doctors, parents of children recovering from autism and more severe childhood neurodegenerative disorders, and individuals who have traveled from around the world to come to the Dr. Goodenowe Restorative Health Center in Moose Jaw to heal from ALS, MS, dementia, and other neurological disorders. Day 1: Open House at the Restorative Health Center Date: July 30, 2024 Time: 12:00-5:00 PM Location: Dr. Goodenowe Restorative Health Center, 1350 Lakeview Rd, Moose Jaw, SK S6H 7T2, Canada Price: Free admission for all interested families The Dr. Goodenowe Restorative Health Center is a one-of-a-kind facility dedicated to restoring health in demyelinating and neurodegenerative health conditions such as ALS, Parkinson's, Multiple Sclerosis, and Dementia. Stop by and learn about Dr. Goodenowe's ground-breaking technologies and how he is changing neuro-degeneration to neuro-restoration and why people from all around the world are coming to Moose Jaw to restore their health. Join us for a day of celebration and community spirit! The festival will feature delicious food and drinks for families, exciting games, fun activities, giveaways, and prizes. Day 2 (12PM-4PM) : Grand Opening of The Dr. Goodenowe Community Health Center Date: July 31, 2024 Time: 12:00 PM - 4:00 PM Location: Town and Country Mall, 1235 Main St N Moose Jaw SK S6h 6M4 Price: Free for All Autistic Residents of Moose Jaw, Aged 2-30 The Dr. Goodenowe Community Health Center is dedicated to working in and with the community of Moose Jaw to restore health and vitality to all residents as part of Dr. Goodenowe's Moose Jaw Vitality Project (MVP). The Restorative Health in Autism program is the Center's inaugural program. Open and free to participate for all children and young adults from age 2 to 30, the Program aims to use science, education, compassion, and love to restore health and function in children, adults, and families struggling with the challenges of autism. Drop in at the Center, meet the team, get some information and sign up! Day 2 (5PM – 8PM): Lecture & Information Night at Connor Park Pavilion Date: July 31, 2024 Time: 5:00 PM Location: Connor Park Pavilion Price: Free Admission We will be officially kicking off the Moose Jaw Restorative Health in Autism Program with an incredible evening featuring a lineup of doctors, parents, and patients with personal knowledge and experience using Dr. Goodenowe's Plasmalogens and Restorative Health Protocols. There will be informational sessions and resources available for families with autism. Sign up for the program and get all your questions answered. Come visit us for specific information and start participating in this one-of-a-kind community health program. Don't miss this chance to be part of a transformative event that combines family fun with cutting edge research. How to Join: In Person: Connor Park Pavilion: Laurel Rd, Moose Jaw, SK S0H 0N0 Virtually: Participate online and be a part of this momentous occasion from anywhere in the world - Click here to Sign up for Virtual Zoom Access Watch Mission Statement from Dr. Goodenowe Here! Come one, come all! Let's celebrate, learn, and support our community together. SOURCE Dr. Goodenowe Research

June 6, 2023. SASKATOON, SASKATCHEWAN, CANADA. Med-Life Discoveries, LP (“MLD”, or the “Company”), is a Canadian biopharmaceutical company dedicated to advancing a therapy to reduce symptoms for a devastating pediatric rare disease, Rhizomelic chondrodysplasia punctata (RCDP), which causes significant physical and neurological delays in children who inherit it. MLD has received a No Objection Letter (“NOL”) from Health Canada to begin a Phase 1 healthy adult trial for lead compound PPI-1011. This is a milestone on the path towards a potential therapy for children with RCDP. MLD’s focus is the development of plasmalogen therapeutics for complex diseases associated with plasmalogen deficiencies. PPI-1011 is a novel, first-in-class synthetic plasmalogen therapy being advanced for the treatment of RCDP. There is currently no treatment for RCDP and life expectancy for children with the disease is short, often no more than 10 years. Children with RCDP experience significant challenges including limited growth, movement and communication, impacting their quality of life and that of their families. The NOL provides MLD with regulatory approval to move forward with a clinical trial in Canada, which will enrol healthy adult volunteers later this month. This first-in-human Phase 1 will evaluate the safety and tolerability of PPI-1011 and represents the first regulatory-approved trial for a pharmaceutical-grade plasmalogen drug of its kind. MLD has been focused for the past six years on the development and advancement of a portfolio of plasmalogen-based compounds for RCDP and other neurodegenerative disorders with underlying compromised plasmalogen biosynthesis. “There is a huge unmet need for legitimate, pharmaceutical-grade plasmalogen therapeutics for patients with RCDP, as well as other neurodegenerative diseases such as Alzheimer’s,” said Dr. Shawn Ritchie, Chief Executive Officer and Chief Scientific Officer of MLD. “Receiving regulatory approval to run the Phase 1 human study for a plasmalogen therapeutic is an exciting and long-awaited advance for patients and their families living with RCDP. For our team of scientists and researchers who are passionate and dedicated about solving the puzzle of RCDP and other plasmalogen deficiencies, it is a double win: an exciting milestone in MLD’s scientific research, but also a chance to improve the lives of children around the world living with this rare and devastating disease.” Dr. Tara Smith, Executive Vice President of Innovative Therapies, added: “We are elated to be the first company in the world to successfully complete formal preclinical safety studies for a synthetic plasmalogen drug candidate. We are proud to now bring this drug into the clinic to test its safety with approval of our regulator, Health Canada. While there is still a lot of work ahead of us, this human trial represents a major step towards a potential treatment for RCDP. There is strong support within the Rhizo community for a regulated, pharmaceutical approach like ours, as evidenced by the number of families enrolled in our on-going Natural History study. We thank all the Rhizo families – we couldn’t advance this treatment without your support.” “This is huge step in our journey to develop a medical therapy for our children and families affected by RCDP,” said Dr. Michael Bober, MD, PhD, Medical Director of the Skeletal Dysplasia Program at Nemours Children’s Hospital, Delaware. Dr. Bober also serves as the Principal Investigator for the RCDP Natural History study. “We see a huge unmet need in the children that we treat in our clinic and that we are studying in the prospective Natural History trial. We very much hope that the Phase 1 is successful so that we can then move towards an interventional trial.” About RCDP Rhizomelic chondrodysplasia punctata (RCDP) is a rare genetic pediatric disease that is caused by mutations in any one of several genes that prevents the biosynthesis of a critical class of phospholipids called plasmalogens. The lack of these lipids in the body is the underlying metabolic cause of the disease. There are currently no treatments for RCDP. Patients with RCDP often have skeletal dysplasia, cataracts and profound growth and developmental delays. The children also exhibit severe neurological deficits, seizures, gastrointestinal and pulmonary issues, negatively impacting their quality of life. In addition, their life expectancy is dramatically reduced, with many patients not living beyond 10 years of age. Survival rates and overall disease severity are directly related to the amount of plasmalogens in their bodies. About MLD MLD is a biopharma company located in Saskatoon, Saskatchewan, Canada which is developing a pipeline of plasmalogen-based drugs, including PPI-1011, for RCDP and other neurological indications. More information available at med-life.ca.