Neuromuscular

The trial is set to read out topline data in the second half of 2023 after completing the target enrollment of 73 patients with Duchenne muscular dystrophy aged 6 to 12 years. The connective tissue growth factor inhibitor has been granted orphan drug, fast track, and rare pediatric disease designations.

Richard Finkel, MD, RAINBOWFISH’s principal investigator and the director of the Experimental Neuroscience Program at St. Jude Children’s Research Hospital, offered his take on the recent FDA decision for risdiplam (Evrysdi; Genentech).

Here's what is coming soon to NeurologyLive®.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is sleep disorders.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending June 3, 2022.

The agency pushed the PDUFA date back citing new data analyses that constitute a significant amendment to the NDA.

The founder and chief medical officer of Omniscient Neurotechnology discussed how the use of machine learning and big data will be critical in revolutionizing our understanding of the brain and mental illnesses.

Data from the RAINBOWFISH study and a large-scale extension, FIREFISH, were the basis for risdiplam’s expanded indication, which now includes babies with SMA under 2 months old.

The founder and chief medical officer of Omniscient Neurotechnology commented on the areas of need with understanding the brain map and treating conditions with multiple pathway crossover. [WATCH TIME: 4 minutes]

Here's what is coming soon to NeurologyLive®.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is World MS Day.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 27, 2022.

David Evans, MBA, shared his experience from this year’s event and spoke about the progress that’s been made in advocating for patients in the last several years at the AAN's Neurology on the Hill conference.

Falls, a common occurrence for patients with spinocerebellar ataxia, were significantly reduced following treatment with troriluzole after 48 weeks.

The founder and chief medical officer of Omniscient Neurotechnology provided insight on the successes using connectomics, including the FDA approval of Quicktome, a digital brain mapping platform. [WATCH TIME: 4 minutes]

Here's what is coming soon to NeurologyLive®.

Despite good tolerability with few drug-related treatment emergent AEs and increased muscle volume, the failure to provide clinically relevant improvements in muscle function led to the discontinuation of ACE-083 for CMT in 2020.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is neuropathic disorders.

Neurology News Network for the week ending May 21, 2022. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 20, 2022.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Matthew B. Harms, MD. [LISTEN TIME: 28 minutes]

The co-CEOs and co-founders of Amylyx Pharmaceuticals discussed their immediate thoughts to the recent FDA AdComm meeting, which voted against evidence of efficacy for AMX0035 in ALS. [WATCH TIME: 3 minutes]

A PK/PD model indicated that a higher concentration of nusinersen may predict an additional 5-point increase in CHOP-INTEND score beyond that observed with the approved 12-mg dose.

In preataxia individuals, serum neurofilament levels increased with proximity to predicted ataxia onset, with significant elevations already present 5 years before onset.

In this episode of Connecting ALS, Dave R. Zook, JD, an advisor with expertise in federal regulatory policy, speaks to the process of making drug coverage decisions for newly approved therapies.