Neuromuscular Disease News

Most individuals with myasthenia gravis were satisfied with their patient journey, but those who were dissatisfied cited issues with diagnostic delays, side effects of treatment and lack of advisement on approved therapies.

Treatment with apitegromab was linked to improved muscle strength and range of motion, while being safe and well-tolerated in those with spinal muscular atrophy types 2 and 3, according to a poster presentation.

Long-term treatment with IV administration of a novel therapeutic for those with myotonic dystrophy type 1 led to significant improvement in muscle strength and control and improved quality of life, data show.

Treatment with viltolarsen at 48 weeks improved pulmonary function in a cohort of pediatric patients with Duchenne muscular dystrophy amenable to exon 53 skipping compared with those receiving standard care.

Treatment with givinostat for ambulatory boys with Duchenne muscular dystrophy led to improved motion and reduced decline of muscle function and strength at 18 months, according to a poster at the 2024 MDA Clinical & Scientific Conference.

Treatment with Zolgensma improved motor function in a cohort of children with spinal muscular atrophy, according to a poster at the 2024 MDA Clinical & Scientific Conference.

For young ambulatory boys with Duchenne muscular dystrophy, treatment with pamrevlumab was nonsuperior to placebo after 52 weeks, according to a poster presentation at the 2024 MDA Clinical & Scientific Conference.

The FDA has granted fast track designation to an investigational peptide-conjugated antisense oligonucleotide delivery system to treat myotonic dystrophy type 1, according to the manufacturer.

The FDA has granted rare pediatric disease designation to an investigational therapy for the treatment of Duchenne muscular dystrophy in those with mutations amenable to exon 44 skipping, according to the manufacturer.

An Indianapolis-area biopharmaceutical company announced it has raised $30 million in Series B financing to advance investigational tyrosine kinase 2 inhibitors for multiple sclerosis as well as dermatologic conditions.