On Feb. 7, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.), announced that the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for viltolarsen, an investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority . . .
In 1950, when the Muscular Dystrophy Association was founded, muscular dystrophy and neuromuscular disease weren’t well known or well understood. But they were, as today, affecting an entire community of kids and adults. And so in 1952, MDA made a move to introduce this community of individuals and families to the world. MDA’s National Ambassador . . .
Australian company Antisense Therapeutics announced encouraging results from its phase 2, open-label clinical trial of the company’s investigational therapy ATL1102 for treating Duchenne muscular dystrophy (DMD). The study met its primary endpoint, which was to assess the safety and tolerability of ATL1102 in patients with DMD who are non-ambulatory (unable to walk independently). After six . . .
Yesterday, Mallinckrodt Pharmaceuticals released a statement to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program. The full statement follows. After careful consideration and despite engaging in diligent efforts to research, develop, and commercialize MNK-1411, Mallinckrodt has made the decision to terminate study MNK14112096 (BRAVE). The study was designed to . . .
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 . . .
On Dec. 12, the US Food and Drug Administration (FDA) granted accelerated approval to golodirsen (Vyondys 53) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. It is the second exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous . . .
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 . . .
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 . . .
