Opinion|Videos|June 24, 2024
Key Players in Transitioning Patients with SMA from Pediatric Care to Adult Health Care
Key opinion leaders (KOLs) further explore the transition of care from pediatric to adult healthcare systems for patients with spinal muscular atrophy (SMA), emphasizing the essential role of multidisciplinary support in ensuring a successful transition.
Advertisement
Episodes in this series
- Who are the necessary team members to help accomplish successful transition from pediatric to adult SMA care?
Advertisement
Related to this article
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 September 18, 2026.
Brian Lin, PhD, discussed the current evidence linking football participation with ALS, the questions that remain unanswered, and how emerging research may help clarify the association.
In honor of Muscular Dystrophy Awareness Month, held annually in September, NeurologyLive® looks back at the decade-long expansion of the DMD treatment landscape and the data that supports each approval.
Marketed as Fayuvi, the 1-time AAV9 gene therapy showed sustained cerebrospinal fluid heparan sulfate drops and developmental gains among pediatric patients with mucopolysaccharidosis type IIIA in prior studies.
Amy Waldman, MD, Medical Director of the Leukodystrophy Center at Children's Hospital of Philadelphia, discusses zilganersen's mechanism, safety profile, clinical considerations, and what the first-ever approval for Alexander disease means for the broader leukodystrophy field.
Results recently published from the phase 2 HIMALAYA trial reported that SAR443820 did not slow functional decline, and was associated with more adverse events and treatment discontinuations in patients with ALS.
A new study reported that tremor and myoclonus affected nearly 7 in 10 patients with chronic inflammatory demyelinating polyneuropathy, with immunotherapy and beta-blockers showing potential benefit for symptom improvement.
Advertisement
Advertisement
Trending on NeurologyLive - Clinical Neurology News and Neurology Expert Insights
1
FDA Approves Levacetylleucine for Ataxia-Telangiectasia
2
FDA Clears Phase 1b/2 Trial of CAR T-Cell Therapy C-CAR168 for Refractory Progressive Multiple Sclerosis
3
FDA Approves UX111, First Gene Therapy for Pediatric Sanfilippo Syndrome Type A
4
Episode 175: Advancing the Ways of Treating Cluster Headache
5