Santhera Pharmaceuticals announced that it has received a $246,000 grant from the U.S. Food and Drug Administration (FDA) to support its ongoing Phase 1 clinical trial evaluating omigapil as a potential treatment for children and adolescents with congenital muscular dystrophy (CMD). The study, called CALLISTO (NCT01805024), is assessing the pharmacokinetics, safety, and tolerability of omigapil in…
Invitae, which specializes in genetic information, has expanded its neurology and cardiology test offerings by adding 11 new panels for genetic diseases. The company has also updated 17 of its neurology panels and eight of its cardiology panels based on recent discoveries in the field of genetics, including tests for muscular dystrophies.
Tivorsan’s lead investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (U.S. FDA). The human recombinant Biglycan (TVN-102), a natural component of muscles, activates a utrophin-based compensatory pathway that can potentially overcome damage caused by the…
New Drug Applications (NDA) for Marathon’s investigational drug deflazacort have been accepted for filing and granted Priority Review by the U.S. Food and Drug Administration (FDA). The NDAs, one for immediate-release tablet formulations and one for an oral suspension formulation, request that deflazacort be approved for the treatment of Duchenne muscular dystrophy…
The first patients have been dosed in PhaseBio Pharmaceuticals‘ part one of a Phase 2a clinical trial for PB1046, a once-weekly vasoactive intestinal peptide (VIP) receptor activator currently in development to treat cardiopulmonary disorders. Part one is designed to assess the effect of PB1046 in adult patients with stable heart failure with reduced ejection fraction…
The Muscular Dystrophy Association (MDA) and the International Association of Fire Fighters (IAFF) are joining forces this summer to support efforts to find much-needed treatments, and possibly a cure, for muscular dystrophy (MD), amyotrophic lateral sclerosis (ALS) and other muscle-degenerative diseases. Members of…
The Child Neurology Foundation (CNF) recently published a statement in the journal Neurology to address the role of neurologists in transferring pediatric patients into the adult healthcare system. The statement was published in July 27 issue under the title “The neurologist’s role in supporting transition to adult health care:…
The National Institute for Health and Care Excellence (NICE) has just issued new guidance recommending Translarna (ataluren) for children older than 5 who have Duchenne muscular dystrophy (DMD). The guidance, titled “Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene,” follows the…
Duchenne UK is launching a new video fundraising and awareness campaign for Duchenne muscular dystrophy (DMD) called ‘The World’s Strongest Boys’. The campaign is already supported by British sports celebrities including Jessica Ennis-Hill and Owen Farrell to help boys living with DMD think like heroes by focusing on…
For the third consecutive year, the Muscular Dystrophy Association‘s nationwide endurance program, the MDA Team Momentum, is revving to run in the Dec. 11, 2016 BMW Dallas Marathon, Half Marathon and Behringer Relay . So far, more than 150 participants on 61 registered teams are geared up already for MDA – even before registrations begins. Since 2014, more…
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