Neuromuscular

The clinical assistant professor of neurology and neurosurgery at the University of Texas spoke about the staggering number of men who forgo seeing physicians for serious conditions and how the clinical community can help address the challenge.

Neurology News Network for the week ending June 22, 2019.

The FDA has approved an sBLA for deflazacort that expands the label to patients aged 2 to 5 with Duchenne muscular dystrophy.

Zilucoplan, also being developed for generalized myasthenia gravis, is expected to enter into a phase 2 clinical trial in IMNM in the second half of 2019, according to Ra Pharma.

The FDA designation was granted to the SK channel modulator after it showed promise in preclinical and phase 1 studies. Cadent Therapeutics is planning a phase 2a trial for the latter half of 2019.

Neurology News Network for the week ending June 1, 2019.

The National Organization for Rare Disorders is home base for more than 300 member organizations that serve not just patients, but providers who care for those with rare disorders, such as SMA and Duchenne muscular dystrophy.

Zolgensma, an adeno-associated virus vector-based, one-time gene therapy administered via intravenous infusion, is the first and only FDA-approved gene therapy for SMA.

The Director of the Multiple Sclerosis and Hereditary Neuropathy Centers at Seton Hall-Hackensack Meridian School of Medicine shared insight into what clinicians should know about Charcot-Marie-Tooth disease.

The chief medical officer and head of Research & Development at Ovid Therapeutics spoke about what prompted the development of this scale to measure improvement for patients with Fragile X syndrome, and possibly other conditions.