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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.

Episode 7 of the AUPN Leadership Minute features Donald S. Higgins Jr., MD, of the VHA; and Justin C. McArthur, MBBS, MPH, of Johns Hopkins University. [WATCH TIME: 4 minutes]

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

Diana Castro, M.D., discusses newborn screening for spinal muscular atrophy (SMA) and suggests how to care for patients after the results are received.

Early recognition and referral of a patient with suspected spinal muscular atrophy (SMA) to a neurologist.

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

Age, sex, EDSS score, and recent methylprednisolone use were all identified as risk factors for developing severe COVID-19.

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.

The duo from Montefiore Medical Center discussed how the advancement of technology has benefitted diagnosing epilepsy and classifying seizures in newborns.

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.

The director of the Institute for Health, Health Care Policy, and Aging Research at Rutgers University discussed his recent findings on assessing evidence for dementia care interventions.

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.

The director and founder of the Sleep Centers of Middle Tennessee discussed the gap between sleep apnea prevalence and testing.

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

Despite showing better composite scores for cognition and ability to perform activities of daily living, a number of the secondary outcomes showed mixed results with the Eli Lilly investigational agent.

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.

Persons with pediatric-onset MS received higher rates of disability benefits, sickness absence days in their youngest age period, and disability pension days in their oldest age period than those without MS.

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.

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