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 . . .
Researchers at Amicus Therapeutics are looking for adolescents (age 12-18) with late-onset Pompe disease (LOPD) to participate in a phase 3 study. The goal of the study is to evaluate the safety and efficacy of ATB200/AT2221, which researchers hope may improve muscle function and respiratory function compared to the standard of care. Participants will be . . .
Researchers at Momenta Pharmaceuticals are looking for adults with generalized myasthenia gravis (gMG) to participate in a phase 2 study. The goal of the study is to evaluate the safety and efficacy of nipocalimab in patients who did not respond to standard of care. This therapy may improve function by lowering the levels of antibodies . . .
On Oct. 2, NS Pharma Inc. (a wholly-owned US subsidiary of Nippon Shinyaku Co. Ltd.) submitted a rolling New Drug Application (NDA) to the US Food and Drug Administration (FDA) for its exon-skipping therapy under development, viltolarsen (previously NS-065/NCNP-01), to treat Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. On Sept. 26, Nippon Shinyaku . . .
This week, Santhera released a letter to the Duchenne muscular dystrophy (DMD) community regarding an update on Santhera’s DMD drug development programs and its partnership with ReveraGen. The full letter follows. Letter to the DMD community from Santhera: Dear Duchenne community, It was great to connect with so many of you at the Parent Project . . .
Researchers at Italfarmaco are looking for participants with Duchenne muscular dystrophy (DMD) to participate in a phase 3 study. The goal of the study is to evaluate the efficacy and safety of givinostat, a drug that belongs to a class called HDAC inhibitors and is believed to induce the muscle repair process, which may prevent . . .
Dr. Perry Mansfield at the SENTA Clinic in San Diego, Calif., is looking for healthy individuals and patients with neuromuscular disease who have documented neurologic pharyngeal muscle dysfunction (dysfunction of the voluntary muscles that form the pharynx) to participate in a pilot study. This study will help to prove the diagnostic consistency of transmembrane electromyography . . .
Biogen announced positive long-term results from its ongoing phase 2 (NURTURE) clinical trial evaluating Spinraza (nusinersen) for treating spinal muscular atrophy (SMA) at the Cure SMA Annual Conference held June 28 through July 1 this year. New data from the NURTURE study demonstrated that after almost four years, infants who were treated with Spinraza before developing symptoms of SMA . . .
The ALS Association, ALS Finding a Cure (ALSFAC), and MDA announced they have jointly awarded a clinical trial grant totaling more than $2.5 million over two-and-a-half years to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital. The principal investigator is Stanley Appel, MD, co-director of Houston Methodist Neurological Institute, chair of . . .
Scholar Rock Holding Corporation announced positive results from its phase 1 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA), at the Cure SMA Annual Conference held June 28 through July 1 this year. Consistent with previously announced interim findings in February, the final results showed positive safety, pharmacodynamic, and pharmacokinetic data in healthy volunteers, supporting . . .