Neuromuscular

In single-ascending dose and multiple-ascending dose studies, no clinically meaningful changes were noted in hematology, chemistry, vital signs, or electrocardiogram parameters.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Angela Genge, MD, FRCPC. [LISTEN TIME: 13 minutes]

Branded as Qalsody, the FDA has granted approval to Biogen and Ionis' tofersen, making it the fourth therapy approved for ALS and the first for SOD1-associated disease.

The director of the Massachusetts General Hospital ALS Care Center talked about CNM-Au8 treatment across three populations, as well as the HEALEY ALS Platform Trial, at the 2023 AAN annual meeting. [WATCH TIME: 5 minutes]

In a subgroup of patients with 300 m to 400 m 6MWD scores in the phase 3 Study 041, ataluren (Translarna; PTC Therapeutics) showed particular improvements from baseline.

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

The medical director of the Rocky Mountain Movement Disorders Center talked about the motivations of the phase 2 study on ANX005 for Huntington disease at the 2023 AAN Annual Meeting. [WATCH TIME: 3 minutes]

FDA-approved for adults with generalized myasthenia gravis in 2016, eculizumab continued to show promising efficacy and safety responses in adolescents with the disease, regardless of on concomitant use of IVIG.

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 the history of the American Academy of Neurology.

Rozanolixizumab has shown promising effects as a potential treatment for myasthenia gravis, as explained by high response rates on MG Activities of Daily Living and other measures.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 21, 2023.

A. Gordon Smith, MD, FAAN, discusses how to assess for treatment success and why the patient perspective is so important in deciding whether to change therapies.

A. Gordon Smith, MD, FAAN, talks about how new agents and emerging data are enabling clinicians to personalize care for patients with myasthenia gravis.

CSL Behring's subcutaneous immune globulin is the first and only immune globulin available in prefilled syringes for patients with primary immunodeficiency or chronic inflammatory demyelinating polyneuropathy.

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

Research that is already underway will be necessary to confirm the safety and efficacy of this class of Rett syndrome therapies, and targeting IGF-1 may be a possibility for treating additional neurological disorders beyond Rett.

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 stroke and cerebrovascular disease.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 14, 2023.

The chief medical officer of QurAlis talked about the potential of QRL-201 in patients with ALS from her talk presented at the 2023 MDA conference. [WATCH TIME: 4 minutes]

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota talked about the potential of investigating inducted pluripotent stem cells in neuromuscular diseases at MDA’s 2023 conference. [WATCH TIME: 5 minutes]

In patients with high C-reactive protein levels treated with NP001, investigators observed a 64% slower rate of decline in forced vital capacity.

A. Gordon Smith, MD, FAAN, discusses the findings and implications of phase 3 ADAPT trial and open-label extension study evaluating the use of efgartigimod in the treatment of patients with myasthenia gravis.

A. Gordon Smith, MD, FAAN, explores the rationale for targeting the neonatal Fc receptor and the mechanisms for efgartigimod for use in the treatment of myasthenia gravis.

The multicenter, multiple-ascending dose trial will feature 64 patients with ALS who will be assessed on safety and pharmacokinetic outcomes.

A. Gordon Smith, MD, FAAN, considers how rituximab and eculizumab fit into the current standard of care for myasthenia gravis.

A. Gordon Smith, MD, FAAN, discusses the role of acetylcholine esterase inhibitors, immunosuppressive agents, IV immunoglobulin, corticosteroids, as well as newer agents in the treatment of myasthenia gravis.

The neuromuscular physician at Virginia Commonwealth University talked about the promising future of gene therapy and access to resources in Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

Sitra Tauscher-Wisniewski, MD, vice president clinical development and analytics, Novartis Gene Therapies, talked about the new data findings from a study on gene therapy Zolgensma at the 2023 MDA conference.

In the phase 2 study, patients with Guillian-Barré syndrome will receive imlifidase on day 1, followed with intravenous immunoglobulin on days 3-7, with outcomes of safety and change in disability.

At the 2023 MDA’s Clinical and Scientific conference, the neurology resident at Penn Medicine talked about the challenges of delivering intrathecal therapies and the importance of planning clinical resources for patients with ALS. [WATCH TIME: 3 minutes]