Neuromuscular

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.

The signs and symptoms leading a pediatrician to a diagnosis of spinal muscular atrophy (SMA) are discussed.

The findings were consistent with the established safety profile for eculizumab in other non-neurologic indications, according to study authors.

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Over a 2-year period, SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores.

Neurology News Network for the week ending March 20, 2021.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending March 19, 2021.

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.

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.

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.