Neuromuscular

Data presented at WORLDSymposium 2021 suggest that the Sanofi enzyme replacement therapy is effective and safe compared to the company’s other agent, alglucosidase alfa.

Both therapeutic programs utilize AAD2-GDNF gene therapy targeted to brain structures vulnerable to Parkinson disease and multiple system atrophy.

The assistant professor of pediatric neurology at the Washington University in St. Louis spoke to the roles of multiple specialists in CP diagnosis.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending February 12, 2021.

The proportion of patients treated with prednisone and the prednisone dose tended to be lower in the rapid-tapering group at 15 months compared to slow-tapering arm.

The treatment can be used in children who do not respond well to anticholinergic medications.

The Endowed Chair in Pediatric Neuromuscular Disorders and professor of clinical pediatrics and neurology, at Children’s Hospital Colorado discussed the future of SMA treatment.

Lisa Belter, MPH, director of research analytics at Cure SMA, discussed a recently published study on the rising costs of living for patients with spinal muscular atrophy.

Wildon Farwell, MD, MPH, shed some light on the ongoing phase 4 trial and what clinical insights might be gleaned from the data.

Here's what is coming soon to NeurologyLive.

The director of the Massachusetts General Hospital ALS Care Center discussed the greatest needs in ALS research and why focusing on genetics can play a big role into understanding the disease course.

The chief scientific officer of QurAlis discussed how QRA-244 is more specific and potent than retigabine, a treatment currently in clinical studies.

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

The director of the Massachusetts General Hospital ALS Care Center spoke on the importance of data-based registries and how they can grow ALS research.

Here's what is coming soon to NeurologyLive.

The director of the Massachusetts General Hospital ALS Care Center discussed the origins of ‘The Data is Here’ campaign and the reason to launch it now.

Neurology News Network for the week ending January 30, 2021.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending January 29, 2021.

The primary investigator of the phase 4 RESPOND study discussed the role that postmarketing studies can play in assessing treatments for SMA.

Brian Frederick, PhD, discusses how the short life expectancy after diagnosis leads many patients with ALS to lose their lives before receiving disability benefits.

Researchers hope that R-loop modulation may hold potential in the future treatment of ALS.

The director of the Massachusetts General Hospital ALS Care Center sat down to discuss ‘The Data is Here’ campaign from Answer ALS, the world’s largest open-sourced ALS data portal.

The primary investigator of RESPOND discussed the trial’s goals and measures, as well as insight into why clinical trials like it are important in spinal muscular atrophy.

Preclinical studies have shown that the homeoprotein hEN1 is essential for the survival and maintenance of spinal cord alpha motor neurons that innervates muscles throughout the body.

Brian Wainger, MD, PhD, assistant professor of neurology and anesthesiology, Harvard Medical School, discussed the recently published phase 2 trial a repurposed epilepsy drug in patients with ALS.