, ,

Sarepta Therapeutics receives Complete Response Letter for golodirsen

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 […]

,

All PolarisDMD sites are now launched

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 […]

Survey for Canadian families living with Duchenne Muscular Dystrophy

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 […]

,

Exondys 51 slows down respiratory decline

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™

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 […]

La Force & Max’s Big Ride in the parliament of Canada

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. […]