A memorable day Monday, June 10 was the day that closed the loop of the event of Max’s Big Ride to End Duchenne Muscular Dystrophy. Our two families are living with Duchenne muscular dystrophy. We share the same battle, that of access to treatment. Yesterday was a memorable day because the government has heard us. […]
La Force DMD is seeking your participation in a research survey for parents or guardians of sons with Duchenne muscular dystrophy and adult men (18+ years) with Duchenne. This is an international survey about your preferences and priorities for Duchenne treatments. Regulatory agencies such as the EMA and FDA rely on the input of patients, […]
27 global sites currently open for enrollment Clinical trial sites in Europe are now open for enrollment in Bristol and Manchester, England; Munich and Hamburg, Germany; Dublin, Ireland; and Gothenburg, Sweden, with additional sites in Europe expected in the coming weeks. This means that PolarisDMD is now active in 7 countries! The Phase 3 PolarisDMD […]
Clinical Trials Simplified and La Force DMD join forces to accelerate the development of new therapies for DMD During a conference about rare diseases, Marie-Catherine Du Berger, president of La Force DMD met Carole Abi Farah, Ph.D., General Manager and Co-founder of Simplified Clinical Trials. Clinical trials provide early access to treatments, contribute to medical […]
Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500 – 5,000. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no […]
– Ambulatory Duchenne patients who are five years and older with a nonsense mutation can now access a treatment that targets the underlying cause of DMD – PTC Therapeutics, Inc. today announced that Translarna™ (ataluren) has been granted marketing approval from the Brazilian National Health Surveillance Agency (ANVISA) under rare diseases procedure, for the […]
The brother movie is the story of the Karouani family in France and their daily life with Kaïs, their son, who has Duchenne muscular dystrophy (DMD). Duchenne is a degenerative muscle disease. In France, it’s called Duchenne myopathy. There is no cure and no hope of recovery. The life expectancy of people with DMD is […]
FibroGen Receives Orphan Drug Designation from the U.S. FDA For Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy FibroGen, Inc. a leading biopharmaceutical company discovering and developing a pipeline of first-in-class therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company’s anti-CTGF antibody, pamrevlumab, for […]
Audentes Therapeutics, Inc., a leading Adeno-associated virus (AAV)* based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases; announced it had expanded its scientific platform and pipeline to advance vectorized antisense treatments for the treatment of Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The Gene Therapy […]
Catabasis is enrolling boys ages 4 to 7 (up to 8th birthday), any mutation type, who have not been on steroids for at least the past six months. What is Edasalonexent? Edasalonexent (CAT-1004 is being developed as a potential foundational disease-modifying therapy for all patients affected by DMD, regardless of their underlying mutation. It […]
