Sarepta Therapeutics receives Complete Response Letter from the US Food and Drug Administration for golodirsen New Drug Application Sarepta Therapeutics, Inc announced it had received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) seeking accelerated approval of golodirsen injection for the treatment of Duchenne […]
Catabasis announces that all 40 sites, across eight countries, are now fully launched for the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy! La Force is happy to share the latest edition of the Catabasis Connection newsletter about the Phase 3 PolarisDMD trial for edasalonexent in Duchenne. The Phase 3 PolarisDMD trial is […]
Dear Duchenne Family, On behalf of Stand for Duchenne Canada, Jesse’s Journey and La Force DMD, thank you for your interest in participating in this survey for Canadian families living with Duchenne Muscular Dystrophy (DMD). The purpose of this survey is to gather vital information to submit to Health Canada, describing the impact Duchenne […]
Thanks to Bio Space, IOS Press, Science Daily for the content of this blog. Duchenne muscular dystrophy is characterized by progressive muscle degeneration. In DMD patients, the pulmonary function becomes progressively impaired as the dystrophic process affects respiratory muscles, including the diaphragm. Strategies to arrest this severe gradual deterioration are needed to extend lives and improve […]
PTC Provides Update on Translarna™ (ataluren) Application for Label Expansion June 28, 2019 > Original press release < PTC Therapeutics, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has completed their review of a proposed indication extension of Translarna™ (ataluren) for the treatment of patients […]
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 […]
