Neuromuscular

A majority of patients across all 3 cohorts maintained or improved their motor function shown by changes from baseline in Revised Hammersmith Scale scores.

A case report showed 1 patient in the vaccine arm developed Guillain-Barré syndrome, though 1 in the placebo arm also developed the syndrome, implying coincidence rather than causation.

Overall, 91% of participants in the pooled bimagrumab group and 89.1% in the placebo group had at least 1 or more treatment-emergent adverse event.

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Take 5 minutes to catch up on NeurologyLive's highlights from the week ending April 2, 2021.

An update on the phase 1b study of Pfizer’s investigational DMD treatment was presented at the MDA 2021 conference.

Researchers conducted a post-hoc analysis of Study 19 to determine hazard ratios of milestone occurrences in amyotrophic lateral sclerosis.

A recent publication describes the findings of two initiatives to evaluate and raise awareness about the impact that SMA has on adolescents and young adults.

Preliminary results from the IGNITE-DMD trial also showed a decreased in creatine kinase levels and improvements on NSAA and PODCI.

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The Adults with SMA Virtual Therapy Program is sponsored by Genentech and the Dhont Family Foundation.

Researchers observed improvements in motor and respiratory function and biomarkers in the investigational treatment group.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending March 26, 2021.

The VBP-LTE study found no evidence of slowed linear growth, insulin resistance, or osteocalcin decreases.

The senior vice president and chief medical officer at Novartis Gene Therapies shared his perspective on the recent clinical trial results for Zolgensma in SMA type 1.

There was very good to excellent level of agreement between the instrumented insoles and the gold-standard reference system.

Two-year data from the SUNFISH study were presented at the MDA 2021 Clinical and Scientific Conference.

Researchers conducted a real-world study of prednisone to deflazacort switch in patients with Duchenne muscular dystrophy and Becker muscular dystrophy.

Assessment of forced vital capacity with treatment of enzyme replacement therapy generally showed some initial stability followed by slow progressive decline.

Closing thoughts on advice to community neurologists treating spinal muscular atrophy (SMA).

Experts remind families of the importance of follow-up visits with the multidisciplinary team during COVID-19.

Resources are discussed, including the Child Neurology Foundation’s telehealth tip sheet, to help families navigate telehealth.

Several spinal muscular atrophy (SMA) resources are suggested to help physicians and families, including Cure SMA and standard of care guidelines.

Discussing the diagnosis of spinal muscular atrophy (SMA) with the family and lining up appointments with the multidisciplinary team to help care for the patient.

Diana Castro, M.D., stresses the value of the initial in-person neurology visit for evaluation of spinal muscular atrophy (SMA) to ensure a correct diagnosis. The next step in the process would be genetic testing.