
Over a 2-year period, SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores.
Over a 2-year period, SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores.
Researchers used predictive swallowing score to help guide decision making in gastric tube placement.
Black patients accounted for 27% of the patients in the COVID-19 CVD Registry but accounted for 31% of the ischemic stroke cases.
Researchers also found that compared to direct to CT patients, DTAS patients has lower onset-to-reperfusion and door-to-groin times.
A greater proportion of patients identified by newborn screening achieved a greater than 4-point increase in CHOP-INTEND scores during the observation period compared to those clinically diagnosed.
The neurologist from Massachusetts General Hospital discussed when to prescribe disease-modifying therapy and some challenges in caring for patients with RIS.
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.
The findings suggest the externally altered proprioceptive feedback in forced exercises may be increasing cortical engagement.
Most treatment-emergent adverse events were mild in severity across both studies, with no evidence of renal toxicity, cardiac signals, or deaths.
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.
The fellow of autoimmune neurology at Massachusetts General Hospital discussed the need for follow-up with patients long-term after TNFα inhibitor treatment.
Researchers conducted a pooled safety analysis of the FIREFISH, SUNFISH, and JEWELFISH studies.
Scholar Rock noted that the topline results from the 52-week treatment period are expected to be announced in the second quarter of 2021.
The postdoctoral researcher at Columbia University detailed a number of topics regarding the impact concealing multiple sclerosis diagnosis can have on a patient.
Ishu Arpan, PhD, senior research associate at Oregon Health & Science University, also discussed further studies she would like to see conducted in MS falls.
LT-001 and LT-002 followed up patients treated with onasemnogene abeparvovec from the START, STRONG, STR1VE, and STR1VE-EU studies.
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.
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.
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.
Treatment with nusinersen demonstrated no clinically meaningful changes in vital signs or clinical laboratory parameters, including urine parameters, in those with spinal muscular atrophy.
Researchers also found that, in comparing ataluren to standard care, ataluren delayed loss in pulmonary function.
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.
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.
In assessing patients treated with siponimod, researchers also saw that plasma neurofilament light levels slightly predicted a reduced risk of disease worsening.
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.
The senior investigator at the National Institutes of Neurological Disorders and Stroke discussed 2 treatments currently being evaluated for use in multiple sclerosis.
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.
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.
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.
The retrospective analysis in patients aged 55 years and older underlines the importance of testing different patient populations.