Latest Conference Coverage

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.

No Greater Risk of Serious COVID-19, Severe Outcomes Linked to Cladribine Treatment

Data provide an update to the post-approval safety profile of cladribine tablets in patients with relapsing MS in reference to COVID-19 infections.

The Need for Guidelines on the Management of Radiologically Isolated Syndrome: Jiwon Oh, MD, PhD

The staff neurologist and medical director of the Barlo Multiple Sclerosis Program at St Michaels Hospital discussed the lack of guidelines for patients with RIS and the research efforts needed going forward to establish them.

MS Relapses, Health Care Utilization Greatest Among COVID-19 Vaccinated Patients Treated With Ocrelizumab, Fingolimod

Previously reported data show patients with multiple sclerosis treated with anti-CD20s have reduced response to COVID-19 vaccines, but there has been limited research on MS outcomes among vaccinated patients.

Differentiating Radiologically Isolated Syndrome and Multiple Sclerosis With Imaging Biomarkers: Jiwon Oh, MD, PhD

The staff neurologist and medical director of the Barlo Multiple Sclerosis Program at St Michaels Hospital discussed her presentation at ACTRIMS Forum 2022 involving imaging biomarkers of RIS/MS prodrome. [WATCH TIME: 4 minutes]

Understanding Frequency, Age, and Sex Associations With Autoimmune Encephalitis Biomarkers: Amy Kunchok, MD

The staff neurologist at Cleveland Clinic’s Mellen Center for Multiple Sclerosis Treatment and Research discussed the results of a new study on age and sex as determinants of autoimmune encephalitis. [WATCH TIME: 3 minutes]

1-Year Disability Progression, Improvement Similar for Patients With Primary, Secondary Progressive MS

Investigators noted the need for more transformational treatments to improve disability for patients with both primary and secondary progressive multiple sclerosis.

Online Adaptive Cognitive Training, tDCS Feasible for Patients With Multiple Sclerosis

Data suggest that there is a greater cognitive benefit for patients with higher levels of baseline neurological disability treated with the training and transcranial direct current stimulation.

Treating Cognitive Deficits in MS With Transcranial Direct Current Stimulation: Leigh Charvet, PhD

The professor of neurology at the NYU Grossman School of Medicine discussed her presentation at ACTRIMS Forum 2022, which showed better cognitive outcomes in MS following treatment with tDCS. [WATCH TIME: 3 minutes]

NeuroVoices: Matthew Schindler, MD, PhD, on Developing a Predictive Model for Chronic Active Lesions in MS

The assistant professor of neurology at the University of Pennsylvania provided insight on a new way to accurately predict chronic active lesion evolution from newly developing MS lesions using 7T MRI.

MS Treatment With Tolebrutinib Uninterrupted in Patients With COVID-19 Infection

The study found that all COVID-19 cases were mild to moderate in severity, and all enrolled patients recovered and remained in the study.

Silent Clinical Progression Predominantly Associated With Cervical Cord Atrophy

Investigators utilized a novel method to accurately capture spinal cord area at C1 vertebral level from legacy brain MRI scans.

Discussing Epstein-Barr Virus and Multiple Sclerosis: Bruce Bebo, PhD

The executive vice president of the National MS Society spoke on recently published data suggesting the relationship between EBV and MS, also sharing his opinion on the potential of vaccines. [WATCH TIME: 2 minutes]

Ganaxolone’s Significant Effect on CDKL5 Deficiency Disorder: Elia Pestana-Knight, MD

The pediatric epileptologist at Cleveland Clinic provided insight on her presentation at the 2022 AAN Annual Meeting that evaluated ganaxolone in phase 3 study of patients with CDKL5 deficiency disorder. [WATCH TIME: 4 minutes]

Patient-Reported Outcomes of MS Fatigue Understudied in Drug Development Trials

Most trials that reported on MS-related fatigue measured its change from baseline and whether that change was statistically significant.

Using Radiologic Markers to Predict Chronic Active Lesion Evolution in MS: Matthew Schindler, MD, PhD

The assistant professor of neurology at the University of Pennsylvania discussed his abstract at ACTRIMS Forum using 7T MRI features of newly developed MS lesions to predict chronic active lesions. [WATCH TIME: 4 minutes]

Taking a More Targeted Approach to Treating Progressive MS Using BTK Inhibition

Regardless of prophylactic or therapeutic paradigms, treatment with evobrutinib resulted in significantly reduced disease severity and improvement of immunopathological parameters of MS disease.