On Oct. 30, AveXis released a letter to the spinal muscular atrophy (SMA) community regarding a decision by the US Food and Drug Administration (FDA) to place a partial hold on clinical trials for intrathecal (IT) administration of AVXS-101. The full letter follows. The order stops enrollment of patients in the high-dose group of AveXis’ . . .
Several biotech and pharma companies presented interim results at the 24th Annual Congress of the World Muscle Society (WMS) held earlier this month in Copenhagen, Denmark. This roundup covers a few of these updates. Catabasis Pharmaceuticals presented positive results from its phase 1/2 MoveDMD trial and open-label extension with edasalonexent in boys with Duchenne muscular . . .
Texas-based Reata Pharmaceuticals announced positive results of its pivotal phase 2 clinical trial (MOXIe) of omaveloxolone in patients with Friedreich’s ataxia (FA). Patients treated with omaveloxolone showed a statistically significant improvement in the modified Friedreich’s Ataxia Rating Scale mFARS (a scale used to measure neurological function) after 48 weeks of treatment compared to placebo, and . . .
In July of this year, Capricor Therapeutics announced positive interim results from its phase 2 (HOPE-2) clinical trial evaluating CAP-1002, the company’s lead investigational therapy for Duchenne muscular dystrophy (DMD), to treat boys and young men in advanced stages of the disease. In that analysis, meaningful improvements in mid-level upper limb and hand function were . . .
Charles Thornton, MD, professor of Neurology at the University of Rochester, was awarded an MDA clinical research network grant (CRNG) totaling $1,118,673 over three years to continue to lead the development of the Myotonic Dystrophy Clinical Research Network, which comprises six medical centers specializing in research and clinical care of myotonic dystrophy (DM) types 1 . . .
Matthew Wood, PhD, professor of Neuroscience at the University of Oxford and director of both the Oxford Rare Disease Center and the MDUK Oxford Neuromuscular Center, was awarded an MDA research grant totaling $210,000 over two years to optimize the delivery of genetic therapies such as oligonucleotides to tissues for treating Duchenne muscular dystrophy (DMD). . . .
At the World Muscle Society annual meeting held earlier this month in Copenhagen, Denmark, ReveraGen announced positive results from its ongoing phase 2 clinical trial of vamorolone for treating Duchenne muscular dystrophy (DMD) in boys. Building upon previously reported positive results, CEO Dr. Eric Hoffman presented data gathered after 18 months of treatment in the . . .
Even though the Martinez family lives in Burbank, Calif., within an hour’s drive to Los Angeles’ Cedars-Sinai Medical Center, their road to the hospital’s specialized clinics — to Dr. Robert Baloh and his work there — was a long one. Daniel and Gladis Martinez have two daughters: Genesis, 15, and Isabella, 14, born just 10 . . .
Martin Childers, PhD, DO, chief medical officer at Asklepios BioPharmaceutical Inc. in North Carolina, was awarded an MDA research grant totaling $192,500 over one year to perform pre-clinical studies using an adeno-associated virus (AAV) to deliver a gene therapy for limb-girdle muscular dystrophy type 2I (LGMD2I). Limb-girdle muscular dystrophies are a diverse group of disorders . . .
Michael Shy, PhD, professor of Neurology and Pediatrics at the University of Iowa, was awarded an MDA Clinical Research Network Grant (CRNG) totaling $423,413 over three years to further develop the Inherited Neuropathies Consortium (INC), a network of clinical investigators dedicated to developing the infrastructure necessary to evaluate therapies for patients with inherited peripheral neuropathies, . . .