Latest Conference Coverage

Determining DMT Timing in Radiologically Isolated Syndrome: Ilena George, MD

The neurologist from Massachusetts General Hospital discussed when to prescribe disease-modifying therapy and some challenges in caring for patients with RIS.

Physical Activity a Useful End Point for Charcot-Marie-Tooth Disease Trials

Researchers found that physical activity as measured by wearable devices was significantly associated with in-person measures of strength and function in patients with CMT.

Forced Exercise Shows Improvement in Poststroke Lower Extremity Motor Function

The findings suggest the externally altered proprioceptive feedback in forced exercises may be increasing cortical engagement.

Casimersen Shows Safe and Effective Profile in Multiple DMD Studies

Most treatment-emergent adverse events were mild in severity across both studies, with no evidence of renal toxicity, cardiac signals, or deaths.

Mobile Stroke Units Superior to Standard Management in 90-Day Stroke Outcomes

Treatment with tPA within 0 to 60 minutes of last known normal occurred for 33% of patients on mobile stroke units, compared to 3% who received standard management.

Investigating Causes of CNS Demyelination: Spencer Hutto, MD

The fellow of autoimmune neurology at Massachusetts General Hospital discussed the need for follow-up with patients long-term after TNFα inhibitor treatment.

Risdiplam Shows Favorable Safety Profile Across SMA Types

Researchers conducted a pooled safety analysis of the FIREFISH, SUNFISH, and JEWELFISH studies.

Apitegromab Demonstrates Safety, Efficacy in Spinal Muscular Atrophy

Scholar Rock noted that the topline results from the 52-week treatment period are expected to be announced in the second quarter of 2021.

NeuroVoices: Anne Kever, PhD, on MS Diagnosis Disclosure, Concealment Behaviors

The postdoctoral researcher at Columbia University detailed a number of topics regarding the impact concealing multiple sclerosis diagnosis can have on a patient.

The Importance of Identifying Those at Risk of Falling in MS

Ishu Arpan, PhD, senior research associate at Oregon Health & Science University, also discussed further studies she would like to see conducted in MS falls.

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.