Neuromuscular Disease News

The FDA has approved a two-part combination therapy in 65 mg capsules to treat adults with late-onset Pompe disease who weigh at least 40 kg and are not improving on their current enzyme replacement regimen.

The FDA’s Cellular, Tissue and Gene Therapies Advisory Committee has voted to deny a biologics license application for NurOwn, an investigational mesenchymal stem cell therapy for the treatment of amyotrophic lateral sclerosis.

PHILADELPHIA — A higher dietary glycemic index through diet modification was associated with both slower functional decline and longer survival in a cohort of individuals with ALS, according to a poster presentation.

The FDA has accepted a new drug application from Neurocrine Biosciences Inc. for its novel granule formulation of oral Ingrezza capsules, for those with tardive dyskinesia or chorea associated with Huntington’s disease.

PHILADELPHIA — Biomarkers are enabling the detection of neurological diseases up to 3 decades before a diagnosis would typically occur, Jane Paulsen, PhD FANA, says in this Healio video.

A global clinical-stage specialty biopharmaceutical company has announced positive topline results from its study of its investigational therapeutic AM0-02 to treat children and adolescents with congenital myotonic dystrophy.

A Southern California-based bioscience firm has announced additional positive data on FDA-approved Ingrezza capsules to treat adults with chorea associated with Huntington’s disease.

A Bay Area-based biopharmaceutical company has announced initiation of a phase 1 clinical trial for its oral, small molecule SARM1 inhibitor, NB-4746, to treat a wide range of conditions affecting the central, peripheral and ocular nervous systems.

The FDA has approved once-daily Ingrezza capsules for adults with chorea associated with Huntington’s disease.

The FDA has granted orphan drug designation to Avidity Biosciences Inc. for AOC 1044, its investigational monoclonal antibody to treat Duchenne muscular dystrophy in those with mutations amenable to exon 44 skipping.