On Dec. 17, Amylyx Pharmaceuticals and the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital announced positive results from the phase 2 (CENTAUR) trial assessing treatment with the company’s investigational therapy, AMX0035, in patients with amyotrophic lateral sclerosis (ALS). The study met its primary endpoint, showing a statistically significant slowing of . . .
Today, Solid Biosciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding updates on a serious adverse event (SAE) experienced by one child dosed last November and new biomarker data from the company’s IGNITE DMD clinical trial. The trial is still on hold pending review by the US Food and Drug Administration (FDA). The full . . .
On Nov. 19, Santhera Pharmaceuticals announced publication of long-term data from its SYROS study showing a reduction of decline in lung function in people with Duchenne muscular dystrophy (DMD) who were treated with idebenone, the company’s experimental therapy for respiratory symptoms associated with DMD. The data from the retrospective study was published in the journal Neuromuscular . . .
Today, Wave Life Sciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program for suvodirsen, its therapy for patients amenable to exon 51 skipping, and its therapy for patients amenable to exon 53 skipping. The full letter follows. Dear Duchenne community: We have a disappointing update . . .
Israel-based Kadimastem Ltd. announced positive interim results from the first cohort of its phase 1/2a clinical trial evaluating the safety, tolerability, and preliminary efficacy of AstroRx in patients with amyotrophic lateral sclerosis (ALS). Results presented at the 7th International Stem Cell Meeting of the Israel Stem Cell Society held recently in Tel Aviv, Israel, showed that patients . . .
At the 24th Annual Congress of the World Muscle Society (WMS) held in October 2019 in Copenhagen, Denmark, Zogenix presented positive results from its pivotal phase 2 retrospective clinical trial (RETRO) testing the safety and efficacy of pyrimidine nucleoside substrate enhancement therapy (SET) MT1621 for treating thymidine kinase 2 (TK2) deficiency. The experimental therapy was developed . . .
On Nov. 19, Santhera Pharmaceuticals released a letter to the Duchenne muscular dystrophy (DMD) community announcing its decision to suspend enrollment of new participants in the BreatheDMD Expanded Access Program (EAP) until the SIDEROS clinical trial is fully enrolled. The full letter follows. SIDEROS clinical trial and BreatheDMD program update: Dear Duchenne community, In February 2018, Santhera . . .
On Nov. 10, Genentech, a member of the Roche Group, announced positive results from the second part of its pivotal phase 2/3 SUNFISH clinical trial assessing risdiplam in patients with spinal muscular atrophy (SMA) types 2 or 3. The study met its primary endpoint, which was change from baseline in the Motor Function Measure 32 (MFM-32) . . .
Today, Solid Biosciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding the decision by the US Food and Drug Administration (FDA) to place the company’s IGNITE DMD clinical trial on hold because the latest child dosed experienced a serious adverse event (SAE). The full letter follows. Letter to the Duchenne Community: Update . . .
Today, Roche Genentech released a letter to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program. The full letter follows. Letter to the DMD community from Roche Genentech: Dear members of the Duchenne community, We are writing to share with you unfortunate news about our clinical development program . . .
