Latest Conference Coverage

Initial Phase 4 RESPOND Results, Omaveloxolone Continues to Perform in FA, Wearable Sensors Safe for FSHD

Neurology News Network for the week ending March 19, 2022.

Assessing the Growing Treatment Landscape for Myasthenia Gravis, Next Steps in Optimization: Henry Kaminski, MD

The chairman of the department of neurology at George Washington University discussed the reasons to be optimistic, but cautiously, about the expanding myasthenia gravis treatment toolbox. [WATCH TIME: 3 minutes]

Initial Safety Findings From Phase 4 RESPOND Study of Nusinersen Announced

After a mean follow-up of 64 days, the most common adverse events were infections and vomiting, with no deaths or reports of post-lumbar puncture syndrome.

Understanding the Newest Therapeutic Advances in Neuromuscular Diseases

Jinsy Andrews, MD, MSc, FAAN, offered her perspective on the ongoing therapeutic boom in neuromuscular disease and some of the critical needs for physicians to keep up with a rapidly progressing treatment paradigm.

Quality Improvement Project Facilitates Preparation, Treatment Recommendations for Adolescents at Risk for Sleep Disorders

MDA 2022 Highlights the Therapeutic Advances for Neuromuscular Diseases: Donald S. Wood, PhD

The president and CEO of the Muscular Dystrophy Association spoke about the highlights of this year’s annual meeting and the recent therapeutic advances for neuromuscular disorders. [WATCH TIME: 3 minutes]

From the Lab to Bedside in Myasthenia Gravis: Henry Kaminski, MD

The chairman of the department of neurology at George Washington University discussed his presentation at MDA 2022 and the value of hearing patient perspective at the conference. [WATCH TIME: 2 minutes]

Managing New Therapies for Neuromuscular Diseases: Jinsy Andrews, MD, MSc

The director of neuromuscular clinical trials at Columbia University Irving Medical Center shared her perspective on the challenges of familiarizing oneself with the influx of novel medications for neuromuscular diseases. [WATCH TIME: 3 minutes]

New MOXIe Extension Trial Results Consistent With Persistent Effect of Omaveloxolone in Friedreich Ataxia

Updates from the open-label, delayed-start period of the pivotal 2-part MOXIe trial (NCT02255435) support previous positive primary end points findings from part 2 of the study.

Vamorolone Demonstrates Continued Safety, Efficacy in DMD Delayed-Start Analysis

Vamorolone, an investigational agent for Duchenne muscular dystrophy, showed an initial disease-modifying effect that was maintained over a follow-up period of 48 weeks.

The Revolution in Limb-Girdle Muscular Dystrophy With Gene Therapies: Nicholas E. Johnson, MD, MSci

The division chief of neuromuscular disorders and vice-chair of research at Virginia Commonwealth University offered his insight into the advances in genetic approaches to LGMD. [WATCH TIME: 2 minutes]

NeuroVoices: Sharon Cohen, MD, FRCPC, on the Diagnostic Potential of Retinal Imaging for Alzheimer Disease

The neurologist and assistant professor at the University of Toronto discussed advantages hyperspectral retinal imaging tools like RetiSpec offer and when clinicians can expect to see them in clinical settings.

Diagnostic Delays and the Importance of the Physician-Patient Relationship: Amy Shinneman

The 2022 MDA National Ambassador and patient with Bethlem myopathy spoke to the critical need to improve diagnosis delays in neuromuscular disorders and effective communication between patients and their physicians. [WATCH TIME: 2 minutes]

Wearable Sensors Feasible, Reliable for Measuring Functional Performance in FSHD

Data may be useful when analyzing disease progression and treatment efficacy in future facioscapulohumeral muscular dystrophy clinical trials.

Casimersen Increases Exon Skipping, Dystrophin Expression in Patients With DMD Amenable to Exon 45 Skipping

The treatment was well tolerated at 48 weeks in patients with DMD with a confirmed mutation amenable to exon 45 skipping.

Where Tolebrutinib and Other BTK Inhibitors Fit in the MS Treatment Landscape: Jiwon Oh, MD, PhD

The staff neurologist and medical director of the Barlo Multiple Sclerosis Program at St Michaels Hospital discussed how investigational BTK inhibitors will be used among an ever-growing MS treatment toolbox. [WATCH TIME: 3 minutes]

Sarepta Details Phase 3 EMBARK Study in DMD

Part B of the 2-part study will have patients previously on placebo receive SRP-9001 for an additional 52 weeks.

The Need to Elevate Patient Voices in Neuromuscular Disorders: Amy Shinneman

The 2022 MDA National Ambassador and patient with Bethlem myopathy spoke to the importance of including the patient perspective at medical meetings to improve the care paradigm in neuromuscular disorders. [WATCH TIME: 3 minutes]

Ravulizumab Generates Positive Phase 3 Data in Myasthenia Gravis With FDA Review Pending

Individuals on ravulizumab demonstrated statistically significant changes in the primary end point of MG-ADL and in secondary end points such as Quantitative Myasthenia Gravis total score.

Long-Term Sustained Benefit Observed in Pompe Treatment Avalglucosidase Alfa

At 97 weeks, avalglucosidase alfa showed continued benefit on measures such as forced vital capacity and 6-minute walk test distance and was successful for patients who switched off algucosidase alfa.

Nusinersen Shows Long-Term Safety, Benefits for Presymptomatic Infants With SMA

Data from the phase 2 NURTURE study of nusinersen (Spinraza; Biogen) suggest that long-term treatment with the agent is beneficial, and point to the importance of newborn screening and early treatment for spinal muscular atrophy.

Newly Approved Daridorexant Demonstrates Safety, Continued Efficacy at 1-Year

The Future of Retinal Imaging to Predict Alzheimer Disease: Sharon Cohen, MD, FRCPC

The neurologist and assistant professor at the University of Toronto provided insight on the clinical use of retinal imaging tools like RetiSpec and the need for further validation of these approaches in Alzheimer disease. [WATCH TIME: 2 minutes]

Outlining Areas of Unmet Need and Next Steps for Research in MS

Speaking on his own presentation on economic burden in MS, Bruce Bebo, PhD, further shared his thoughts on areas of unmet need and the potential relationship of Epstein-Barr virus and MS.

Serum GFA Identified as a Biomarker for NMOSD, sNfL as a Predictor of Poor Post-Attack Outcomes

A systematic literature review of randomized controlled trials in neuromyelitis optica spectrum disorder was performed, with 7 studies identified and 2 used in analyses.

Autologous Hematopoietic Stem Cell Transplantation in Multiple Sclerosis Is Safe, Efficacious Over Long-Term

Investigators noted the long-term cost-effectiveness and safety of this approach, as it does not require the regular use of disease-modifying therapies.

Muscular Dystrophy Association: A Hybrid Conference Featuring the Latest Advances in Neuromuscular Research, Therapy, and Clinical Care

The Muscular Dystrophy Association's annual meeting will take place in-person and virtually in Nashville, Tennessee, on March 13-16, with more than 950 in-person attendees and 130 presenters.

Overview of Tolebrutinib’s Positive 18-Month Data in Relapsing MS: Jiwon Oh, MD, PhD

The staff neurologist and medical director of the Barlo Multiple Sclerosis Program at St Michaels Hospital provided insight on the tolebrutinib’s mechanism of action, and new data presented at ACTRIMS Forum 2022. [WATCH TIME: 4 minutes]

Addressing ‘Staggering’ Economic Burden for Patients With Multiple Sclerosis

Bruce Bebo, PhD, outlined findings from a recent analysis presented at ACTRIMS Forum 2022, with investigators concluding costs associated with the disease have previously been underestimated.

NeuroVoices: Jiwon Oh, MD, PhD, on Radiologically Isolated Syndrome as a Precursor to MS

The staff neurologist and medical director of the Barlo Multiple Sclerosis Program at St Michaels Hospital provided insight on the use of biomarkers to improve management of RIS and uncover more as it relates to MS.