FDA Issues Complete Response Letter for Vanda Pharmaceuticals' Tasimelteon for Insomnia
March 11th 2024In recent news, the FDA has issued a complete response letter for Vanda Pharmaceuticals' tasimelteon for treating insomnia because of identified deficiencies and cannot be approved in its current form.
Adding to the Literature of KIF5A in ALS Pathogensis: Devesh Pant, PhD
March 11th 2024The instructor in the department of cell biology at Emory University provided context on a presentation at MDA 2024 regarding mutations in Kif5a in familial forms of amyotrophic lateral sclerosis. [WATCH TIME: 4 minutes]
Impact of Indole 3-Lactate Supplementation in Multiple Sclerosis Animal Models: Larissa Jank, MD
March 10th 2024The postdoctoral researcher in the department of neurology at Johns Hopkins School of Medicine discussed findings from a study on the impact of indole 3-lactate supplementation in animal models of multiple sclerosis. [WATCH TIME: 4 minutes]
Amylyx Mulls Over Pulling AMX0035 Following Disappointing Phase 3 PHOENIX Findings
March 8th 2024Over the coming weeks, Amylyx will undergo heavy discussions with regulators and community members to decide the next best steps for AMX0035 after the agent failed to meet its primary end point in its latest study.
Addressing Diversity Gaps in Neuromyelitis Optica Spectrum Disorder Research: Mirla Avila, MD
March 8th 2024The director of the Comprehensive Care MS Center at Texas Tech University Health Sciences Center talked about findings from a subgroup analysis of Hispanic or Latin patients from the N-MOmentum trial presented at ACTRIMS Forum 2024. [WATCH TIME: 3 minutes]
Switching From Anti-CD20 Therapy to Ublituximab Shows Promising Results in Phase 3b ENHANCE Trial
March 7th 2024New data from the phase 3b ENHANCE trial presented at ACTRIMS Forum 2024 demonstrated the smooth transition from intravenous anti-CD20 therapy to ublituximab in patients with multiple sclerosis.
Real-World Effectiveness and Safety of Monoclonal Antibodies in NMOSD: Amy Kunchok, MD, PhD
March 7th 2024The staff neurologist at the Mellen Center for Multiple Sclerosis at Cleveland Clinic talked about findings from a real-world study assessing novel monoclonal antibodies in patients with NMOSD presented at ACTRIMS Forum 2024. [WATCH TIME: 4 minutes]
Intravenous Zolgensma Shows Motor Function Improvement in Heavier Pediatric Patients With SMA
March 7th 2024Findings from the recent phase 3b SMART trial affirm the safety and efficacy of intravenous onasemnogene abeparvovec (Zolgensma; Novartis) in spinal muscular atrophy when patient weights range from 8.5 kg to 21 kg.
Respiratory Patterns Differ in Pediatric Duchenne Muscular Dystrophy
March 6th 2024A recent study presented at MDA 2024 highlighted the evolving respiratory patterns in pediatric patients with Duchenne muscular dystrophy, offering crucial insights for effective respiratory management in this patient population.
Use of the ActiMyo Sensor to Assess 95th Centile of Stride Velocity: Laurent Servais, MD, PhD
March 6th 2024The professor of pediatric neuromuscular diseases at the University of Oxford provided insight on a study presented at MDA 2024 assessing the use of a magneto-inertial sensor in ambulant children with Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]
Integrating a Business Methodology to Improving Care for ALS: Brooke Eby
March 6th 2024At MDA 2024, Brooke Eby shared her perspective as a patient with ALS and the ways to incorporate business approaches to improving clinical care and increasing enrollment for drug trials. [WATCH TIME: 5 minutes]
Preoperative Disease-Modifying Agents Reduce Postoperative Complications in SMA Scoliosis Surgery
March 5th 2024A recent study presented at MDA 2024 suggests that preoperative use of disease-modifying agents for patients with spinal muscular atrophy leads to less severe postoperative complications following scoliosis surgery.
Mechanism of Action of Investigational Agent NMD670 for SMA: Jorge Quiroz, MD, MBA
March 5th 2024The chief medical officer of NMD Pharma shed light on a newly announced phase 2 study assessing NMD670, a neuromuscular transmission enhancer, for patients with spinal muscular atrophy. [WATCH TIME: 4 minutes]
Viltolarsen Shows Significant Pulmonary Function Gains in Ambulant and Nonambulant DMD
March 5th 2024In a recent phase 2 trial analysis of viltolarsen presented at MDA 2024, findings showed improvement in forced vital capacity compared with standard care in patients with Duchenne muscular dystrophy.