The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Mallinckrodt’s investigational new drug (IND) application for Synacthen Depot to treat Duchenne muscular dystrophy (DMD). The invesigational drug is a depot formulation (a sustained-action drug formulation that allows slow release and gradual absorption) of Synacthen (tetracosactide), a synthetic…
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…
Idera Pharmaceuticals recently presented pre-clinical data regarding the gene-silencing mechanisms of its third generation antisense (3GA) technology platform, a potential therapeutic for diseases that include, but are not limited to, facioscapulohumeral muscular dystrophy (FSHD). The presentation, “Precise excision of targeted RNA by third generation antisense (3GA) oligonucleotides” was made at the…
Summit Therapeutics reports that a new formulation of ezutromid (called F6), its Duchenne muscular dystrophy (DMD) treatment candidate, achieved a greater than six-fold increase in maximum plasma levels in DMD patients participating in a Phase 1 clinical trial, compared to results achieved with its current formulation (F3), and at a lower…
Replenishing levels of a molecule naturally occurring in the body called NAD+ could restore muscle function according to scientists at the University of Pennsylvania.
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…
Bamboo Therapeutics has been fully acquired by Pfizer, considerably expanding that company’s holdings in gene therapy. With the acquisition, Pfizer gains several potential therapeutic agents for rare diseases, including one for Duchenne muscular dystrophy (DMD), soon to enter clinical testing; and one Phase 1/2 clinical stage agent, a potential best-in-class recombinant Adeno-Associated Virus (rAAV)…
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