Latest Conference Coverage

Zolgensma Shows Safety and Durability of Efficacy in Long-Term Follow-Ups for SMA Type 1

LT-001 and LT-002 followed up patients treated with onasemnogene abeparvovec from the START, STRONG, STR1VE, and STR1VE-EU studies.

COVID-19 and Muscular Dystrophies, and R&D for Rare Disorders: Sharon Hesterlee, PhD

The executive vice president and chief research officer of the MDA offered her perspective on the ongoing conversations about and impact of the pandemic on the care of patients with muscular dystrophies.

Swallowing Function Possibly Nonissue With Nusinersen in Presymptomatic SMA

At each timepoint, all assessed participants with 3 SMN2 copies and the majority with 2 SMN2 copies were identified by PASA as not being tube fed.

Identifying and Diagnosing Genetic Leukodystrophies as MS Mimics

Alise Carlson, MD, a resident at Cleveland Clinic, discussed her presentation from ACTRIMS Forum 2021 on the misdiagnosis of adult-onset genetic leukodystrophies as multiple sclerosis.

Nusinersen Safety Demonstrated in DEVOTE Phase 2/3 Study in SMA

Treatment with nusinersen demonstrated no clinically meaningful changes in vital signs or clinical laboratory parameters, including urine parameters, in those with spinal muscular atrophy.

Ataluren Delays Loss of Ambulation in Patients With Nonsense Mutation DMD

Researchers also found that, in comparing ataluren to standard care, ataluren delayed loss in pulmonary function.

High-Dose Risdiplam Results in High Percentage of Event-Free Infants With SMA

The high-dose cohort in part 1 of the FIREFISH study also showed that more than 50% of infants could sit without support and more than 90% could feed orally.

Understanding Effects of Diagnosis Disclosure in MS: Anne Kever, PhD

The postdoctoral researcher at Columbia University discussed her abstract from ACTRIMS Forum 2021 on the associations of MS diagnosis disclosure and concealment with anxiety and depression.

Early Plasma GFAP Lowering Predicts Reduced Disability Worsening Risk in Progressive MS

In assessing patients treated with siponimod, researchers also saw that plasma neurofilament light levels slightly predicted a reduced risk of disease worsening.

Muscular Dystrophy Association Virtual Conference to Feature Latest Advances in Neuromuscular Therapy and Clinical Care

Sharon Hesterlee, PhD, executive vice president and chief research officer, MDA, shares an early look at the MDA's 2021 Virtual Clincial and Scientific Conference, March 15-18.

Anakinra and Tolebrutinib for Chronic Inflammation in MS: Daniel Reich, MD, PhD

The senior investigator at the National Institutes of Neurological Disorders and Stroke discussed 2 treatments currently being evaluated for use in multiple sclerosis.

What to Expect from the 2021 MDA Clinical and Scientific Conference: Sharon Hesterlee, PhD

The executive vice president and chief research officer of the MDA gave an overview of the upcoming virtual annual meeting and her insight on what to look for.

Next Steps in Improving Autoimmune Encephalitis Care

The assistant professor of neurology at Mayo Clinic detailed the areas of autoimmune encephalitis research that need more attention, as well as the diagnostic potential of autoantibody assays.

Investigating the 1-Hour Post-Infusion Monitoring Period: Yujie Wang, MD

The neurologist from the University of Washington Medical Center discussed how the study of natalizumab adverse events came about and the importance of this monitoring period.

Older Patients With MS Face Additional Risks With Ocrelizumab Treatment

The retrospective analysis in patients aged 55 years and older underlines the importance of testing different patient populations.

Interleukin-1 Receptor Antagonism and Multiple Sclerosis: Stephanie Blandford, MSc

The PhD candidate at the neuroimmunology laboratory at Memorial University of Newfoundland discussed further research she would like to see conducted with IL-1RA in MS.

African American Patients Have Higher MS Disability Scores Than Hispanic, Caucasian Patients

Researchers found that, in an exploratory analysis of 150 patients, African American patients with MS were most likely to become disabled and lose employment.

Identifying Those at Risk of Falling in Multiple Sclerosis

Ishu Arpan, PhD, senior research associate at Oregon Health & Science University, discussed her team’s investigations into identifying patients with MS at risk of falling.

Fatigue Severely Impacts Quality of Life, Work Productivity in MS

Patients having a relapse, confirmed disability accumulation, or worsening in fatigue had significant negative impact on each of the work productivity and activity impairment measures.

Prescribing DMT in Radiologically Isolated Syndrome: Ilena George, MD

The neurologist from Massachusetts General Hospital discussed physician rationales behind prescribing DMT use in RIS.

Multiple Sclerosis Care Professionals Satisfied With Clinical Video Telemedicine Visits

Researchers found that the percentage of telemedicine visits increased from 15% to 72.8% during the COVID-19 pandemic.

Electronic Pill Bottle Improves DMT Adherence, Subcutaneous Cladribine Personalized Dosing Well-Tolerated, MS Fatigue Unchanged Long-Term

Neurology News Network for the week ending March 6, 2021.

Understanding the Momentum in the Autoimmune Encephalitis Research Community

The assistant professor of neurology at Mayo Clinic discussed his presented talk at ACTRIMS Forum 2021 on autoimmune encephalitis.

African American Patients With MS Switch to Ocrelizumab Treatment Earlier than Caucasian Patients

Researchers also found that patients with progressive MS had longer disease duration before starting DMTs than those with relapsing MS.

How COViMS Registry Is Clarifying MS COVID-19 Risks: Amber Salter, MPH, PhD

The assistant professor of biostatistics at Washington University in St. Louis discussed what role the COViMS registry can play for the MS community at this stage in the pandemic.

Human Oligodendrocyte Differences Due to Environmental Signals Rather Than Lineage

Researchers used single-cell RNA sequencing on adult, pediatric, and infant surgical tissues to study oligodendrocyte types in order to better understand multiple sclerosis.

High Rates of MS Relapse after TNFα Inhibitor Discontinuation: Spencer Hutto, MD

The fellow of autoimmune neurology at Massachusetts General Hospital discusses the ongoing question about whether the demyelination is an unmasking of disease or consequence of the treatment.

Predicting MS Disability With IL-1 Receptor Antagonist Levels: Stephanie Blandford, MSc

The PhD candidate at the neuroimmunology laboratory at Memorial University of Newfoundland discussed her team’s investigations into the use of IL-1RA as a biomarker for MS disability.

NeuroVoices: Dylan Rice, on Improving Adherence to MS DMTs

The clinical research coordinator at Massachusetts General Hospital discussed his presentation from ACTRIMS 2021 on electronic pill bottle monitoring to promote MS medication adherence.

Expanding Research in Autoimmune Encephalitis: Divyanshu Dubey, MBBS

The assistant professor of neurology at Mayo Clinic provided his thoughts on the next steps in understanding autoimmune encephalitis and the direction that research must pivot to.