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Research Updates

First Patient Enrolled in Phase 2 Trial of MNK-1411, Mallinckrodt’s Investigational Therapy for DMD

The first patient has been enrolled in a Phase 2 trial evaluating the effectiveness and safety of MNK-1411, an investigational therapy for Duchenne muscular dystrophy (DMD), announced Mallinckrodt Pharmaceuticals, the treatment’s developer. The company also reported that MNK-1411 was granted orphan medicinal product status by the European Medicines Agency, facilitating the development and eventually the approval process for the treatment candidate. The therapy was given orphan drug designation in […]


A protein could be key to preserving heart function in Duchenne muscular dystrophy

SAN ANTONIO, TEXAS, Aug. 1, 2018 — A protein known to drive nerve cell survival in the brain and spinal cord might also protect failing hearts in children and young adults with Duchenne muscular dystrophy, according to preliminary research presented at the American Heart Association’s Basic Cardiovascular Sciences Scientific Sessions, a premier global exchange of […]


FDA Places Hold on Phase 1/2 Trial of Sarepta’s Gene Therapy for DMD

A Phase 1/2 trial evaluating Sarepta’s microdystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD) was placed on clinical hold by the U.S. Food and Drug Administration. The FDA found trace amounts of plasmid DNA in the raw material used to make the drug. The fragments don’t seem harmful in preliminary tests and the dosing of patients by the end of […]


Consortium.AI to Advance New Treatments for Duchenne Using Artificial Intelligence

Insilico Medicine and A2A Pharmaceuticals, two leading artificial intelligence (AI) biotechs, are collaborating to create a new company called Consortium.AI, which will advance AI for the development of new molecules to treat Duchenne muscular dystrophy (DMD) and other orphan diseases. Consortium.AI will feature computational new candidates designed to target specific diseases that were validated through Insilico’s AI systems. A2A Pharmaceuticals […]


4 Approaches to Treating Duchenne Muscular Dystrophy Highlighted at PPMD Conference

Discussions of the work being done on four different approaches to treating Duchenne muscular dystrophy (DMD) — repairing or replacing dystrophin, managing inflammation, improving muscle mechanics, and editing dysfunctional genes using CRISPR/CAS9 — were a highlight of sessions at the recent 2018 Parent Project Muscular Dystrophy (PPMD) conference in Scottsdale, Arizona. Patients, parents, doctors, researchers, and industry executives […]


PTC Therapeutics Announces Publication of Data Demonstrating the Clinical Differentiated Benefit of Deflazacort

– Post hoc analysis of the placebo arm of ACT DMD Data Published in Muscle & Nerve – SOUTH PLAINFIELD, N.J., July 24, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the publication of data in Muscle & Nerve comparing the efficacy and safety of deflazacort and prednisone/prednisolone from the placebo arm of the ACT DMD study.  The results […]


PTC Therapeutics Announces Positive Data from its Translarna™ Phase II Clinical Trial in Children as Young as Two Years with Nonsense Mutation Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, N.J., July 9, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced the presentation of data from the Translarna (ataluren) Phase II Study 030 demonstrating that the safety and pharmacokinetic profile of Translarna in children from two to five years with nonsense mutation Duchenne muscular dystrophy (nmDMD) was consistent with that for older children.1  Importantly, the […]


Potential DMD Therapy Now in Phase 1 Trial, WVE-210201, Likely To Be Named Orphan Drug in EU

The Committee for Orphan Medicinal Products (COMP), an arm of the European Medicines Agency (EMA), is recommending that WVE-210201 be designated an orphan drug as a potential Duchenne muscular dystrophy (DMD) treatment, its developer, Wave Life Sciences, announced. WVE-210201 is an investigational compound of a type of therapy called exon skipping, which produces shorter but functional proteins. It is currently in a […]


Catabasis Pharmaceuticals Announces Plans for Edasalonexent Phase 3 POLARIS DMD Trial in Duchenne Muscular Dystrophy

CAMBRIDGE, MA, July 9, 2018 – Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced plans for the Phase 3 POLARIS DMD trial with edasalonexent in patients with Duchenne muscular dystrophy (DMD). Catabasis plans to initiate the global POLARIS DMD trial in the second half of 2018 with top-line results expected in the second […]