Neuromuscular

Rachele Berria, MD, PhD, Medical Affairs at Chiesi Global Rare Diseases, discussed the clinical data and implications of newly approved every-4-week dosing of pegunigalsidase alfa for Fabry disease in Europe.

BridgeBio Pharma has submitted a new drug application to the FDA for BBP-418 for the treatment of limb-girdle muscular dystrophy type 2I/R9, supported by interim phase 3 trial data.

The FDA has approved a higher-dose regimen of nusinersen for spinal muscular atrophy, supported by DEVOTE data showing improved motor outcomes, reduced neurodegeneration markers, and a safety profile consistent with prior dosing.

Here's some of what is coming soon to NeurologyLive® this week.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 27, 2026.

Wolfgang Singer, MD, a professor of neurology at Mayo Clinic, spoke about the current state of biomarker research in MSA.

The founder and CEO at the Hereditary Neuropathy Foundation discussed how learning about patient experiences has influenced research in Charcot-Marie-Tooth disease. [WATCH TIME: 5 minutes]

The FDA granted accelerated approval to tividenofusp alfa for Hunter syndrome, introducing the first enzyme replacement therapy designed to cross the blood-brain barrier and target neurologic disease.

The chief medical officer at Edgewise discussed long-term MESA data showing sustained functional stabilization with sevasemten in Becker muscular dystrophy and its potential as a disease-modifying therapy.

Experts discussed new biomarker data from a phase 1/2 study, presented at the 2026 MDA Conference, of the gene therapy SGT-003 in boys living with Duchenne muscular dystrophy.

The 2026 MDA National Ambassador discussed the importance of connecting patients’ experiences with scientific understanding, as well as fostering a sense of community among those living with neuromuscular disease. [WATCH TIME: 2 minutes]

In a plain language summary of the ADHERE trial, subcutaneous efgartigimod improved disability and reduced relapse risk by 61% in CIDP, with sustained benefit and a favorable safety profile.

The chief research officer of the Muscular Dystrophy Association spoke on highlights and themes from the organization's 2026 Clinical and Scientific Conference. [WATCH TIME: 9 minutes]

In episode 1, neurologist Catherine Lomen-Hoerth, MD, PhD, is joined by Timothy Miller, MD, PhD, to discuss pivotal long-term data showing tofersen's meaningful impact in patients with SOD1 ALS.

Here's some of what is coming soon to NeurologyLive® this week.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 20, 2026.

The research portfolio director at the Muscular Dystrophy Association highlighted the collaborative spirit, the expanding therapeutic pipeline, and the growing emphasis on patients’ voices in neuromuscular disease on display at MDA 2026. [WATCH TIME: 8 minutes]

The child neurologist at Children’s Hospital of Philadelphia discussed the real-world safety data of givinostat use in patients with Duchenne muscular dystrophy at the 2026 MDA Conference. [WATCH TIME: 5 minutes]

Early BB-301 gene therapy data show durable swallowing gains and benign safety in OPMD dysphagia, hinting at disease modification.

A phase 1/2 study aims to assess the safety and tolerability of nivudirsen, also known as BMN 351, in single ascending and multiple ascending doses among patients living with Duchenne.

A neurologist at Georgetown University, MedStar Health, discussed phase 2/3 data presented at the 2026 MDA Conference that supports efgartigimod as a novel treatment for inflammatory myopathies. [WATCH TIME: 2 minutes]

Here's some of what is coming soon to NeurologyLive® this week.

A retrospective cohort study reported that myocardial fibrosis detected by cardiac MRI was associated with higher fatigue scores among women carriers of Duchenne or Becker muscular dystrophy.

At the 2026 MDA Conference, a neurologist at Nemours Children's Hospital discussed practical strategies for navigating insurance to ensure patients can access newly available neuromuscular therapies. [WATCH TIME: 3 minutes]

In a retrospective cohort of 33 patients with chronic inflammatory demyelinating polyneuropathy, variants accounted for nearly two-thirds of cases, while intravenous immunoglobulin demonstrated high response rates across subtypes.