Rare Disease Report


The FDA has granted Rare Pediatric Disease Designation to Myonexus Therapeutics for its MYO-101, which is an AAV-based gene therapy for the treatment of limb girdle muscular dystrophy (LGMD) type 2E.
FDA grants orphan drug designation to Sarconeos for Duchenne muscular dystrophy (DMD).
Late Friday afternoon, Novartis announced that the U.S. FDA approved fingolimod (Gilenya) to treat relapsing multiple sclerosis (MS) in children and adolescents age 10 years and older.
This morning, it was announced that Invitae Corporation and Sarepta Therapeutics will be expanding its partnership to continue assisting clinicians in identifying patients with Duchenne muscular dystrophy (DMD).
Cytokinetics, Incorporated and The ALS Association continue their partnership in an effort to fight ALS.
This morning, Acceleron Pharma announced that the US FDA has granted Fast Track designation to ACE-083 for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD).
This morning, Capricor Therapeutics, Inc. announced that the HOPE-2 clinical trial has been initiated at UC Davis Medical Center in Sacramento, CA.
Data from Type 1 spinal muscular atrophy (SMA) treatment suggests efficacy and tolerability.  
At the 70th AAN Annual Meeting this morning Summit Therapeutics plc presented new 24-week interim data from PhaseOut DMD, its Phase 2 trial of ezutromid in Duchenne.
The first patient was enrolled in a Phase 3 clinical trial of amifampridine phosphate (Firdapse) in patients with mmuscle-specific kinase (MuSK) antibody positive myasthenia gravis (MuSK-MG).
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