Neuromuscular

The FIREFISH clinical trial by PTC Therapeutics is 1 of several studies underway evaluating risdiplam in SMA.

The recommendation stems from recently published topline data which demonstrated a clinically meaningful treatment response compared to placebo but failed to meet statistical significance.

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

The antisense oligonucleotide casimersen will be branded as Amondys 45 and is currently being assessed in the ESSENCE phase 3 clinical trial (NCT02500381).

Advancements in gene therapy approaches have opened the door for a glimpse into the future of care for a number of diseases.

The assistant professor of pediatric neurology at the Washington University in St. Louis spoke to the insights of a recent survey she and colleagues conducted.

The director of the Massachusetts General Hospital ALS Care Center detailed the role genetic backgrounds can have on outcomes within ALS clinical trials.

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The director of the Massachusetts General Hospital ALS Care Center discussed some of the biggest challenges for patients with ALS and what should be prioritized in research.

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

Overall, variants within known ALS-linked genes are of potential clinical importance for 42% of patients, according to the study authors.

Scholz was recognized for her application of advanced genetic techniques to the study of neurodegenerative disorders, including dementia with Lewy bodies, multiple system atrophy, frontotemporal dementia, progressive supranuclear palsy, and corticobasal degeneration.

The DMD treatment pipeline boasts a number of therapeutics with varying mechanisms of action and delivery systems, pointing to a promising future for treatment.

Researchers found that 93% of patients with AS exhibited dystonia in their limbs, mouth, neck, or trunk.

With promising data presented at WORLDSymposium 2021 and a PDUFA date looming in May, investigator Priya S. Kishnani, MD, offered her insight on Sanofi’s investigational therapy.

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The assistant professor of pediatric neurology at Washington University in St. Louis spoke to the results of a recent survey she and colleagues conducted.

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.