The NeurologyLive® neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.
August 29th 2025
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 29, 2025.
Myotonic Dystrophy Agent AOC 1001 Demonstrates Successful Targeted Delivery of RNA
December 15th 2022AOC 1001, an agent consisting of a proprietary monoclonal antibody that binds to the transferrin receptor 1, was safe, tolerable, and showed significant reductions in DMPK, a disease-related mRNA.
Building the Biomarker Panel in Neurology: Glial Fibrillary Acidic Protein
December 9th 2022As the treatment paradigm for neurologic diseases rapidly progresses, the need for more thorough biomarker tools to measure disease progression and severity has increased, and in recent years, GFAP has emerged as a valuable candidate to add to the existing panel.
DNL343 to be Assessed in HEALEY ALS Platform Trial Following Positive Phase 1 Findings
December 6th 2022After showing robust inhibition of biomarkers associated with integrated stress response, the eIF28 modulator will be assessed alongside several other potential agents in the HEALEY ALS Platform trial.
The Expectations for the 2023 MDA Conference: Donald S. Wood, PhD; Sharon Hesterlee, PhD
December 1st 2022The president and CEO, as well as the executive vice president and chief research officer, of MDA, together offered an in-depth overview of what to expect from the 2023 MDA Conference, which will be held from March 19-22, 2023, in Dallas, Texas. [WATCH TIME: 12 minutes]
Expanding the Capabilities of Treating Neuromuscular Poststroke Symptoms: Wayne Feng, MD, FAHA
November 29th 2022The division chief of stroke and vascular neurology at Duke Health provided perspective on the multidisciplinary team needed to treat neuromuscular poststroke symptoms, as well as exciting innovations on the horizon. [WATCH TIME: 4 minutes]
Expanding Neuropalliative Care to New Disease States: Benzi Kluger, MD, MS
November 28th 2022The president of the International Neuropalliative Care Society detailed potential disease states that could benefit from neuropalliative care as well as the importance of the patient community in expanding this type of approach. [WATCH TIME: 4 minutes]
NeuroVoices: Benzi Kluger, MD, MS, on the Expansion and Growth of Neuropalliative Care
November 23rd 2022The president of the International Neuropalliative Care Society provided perspective on the growth of the field of neuropalliative care, the organization’s role, and how clinicians are overcoming current barriers in place.
Growth of Neuropallaitive Care and the Bright Future: Benzi Kluger, MD, MS
November 22nd 2022The president of the International Neuropalliative Care Society discussed the area in which neuropalliative has expanded and improved, as well as the potential in the coming years. [WATCH TIME: 2 minutes]
Post-Conference Perspectives and Updates in Neuropalliative Care: Benzi Kluger, MD, MS
November 21st 2022The president of the International Neuropalliative Care Society provided an overview of the organization’s recently concluded conference and the significance of the growth of neuropalliative care. [WATCH TIME: 6 minutes]