Levacetylleucine (AQNEURSA) is approved for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia weighing at least 15 kg, in addition to its existing indication for ...

"Unmet Needs and Mechanisms Behind Newer Agitation Therapies" takes up what still remains unmet in agitation care, and the biology behind a newer treatment option.

The 2026 AHA/ASA adult stroke rehabilitation guideline updates the 2016 framework, addressing evidence-based recovery ...

EPOCH is a single-center, prospective, randomized, open-label, blinded-endpoint (PROBE) trial being conducted at National Taiwan University Hospital. The study was registered in March 2024, and ...

HIMALAYA was a multicenter, randomized, double-blind, placebo-controlled phase 2 trial conducted at 63 sites across 13 ...

The FDA has approved UX111 (Fayuvi; Ultragenyx Pharmaceutical), an adeno-associated virus serotype 9 (AAV9) gene therapy, for the treatment of pediatric patients with Sanfilippo syndrome type A ...

Foundational health behaviors reduce migraine burden, but frequent or disabling attacks commonly require preventive treatment ...

In early September, the FDA approved zilganersen injection (Zanvastro; Ionis) for the treatment of Alexander disease in pediatric and adult patients, marking the first approved th ...

Results recently published from the phase 2 HIMALAYA trial reported that SAR443820 did not slow functional decline, and was associated with more adverse events and treatment discontinuations in ...

Ahead of Myotonic Dystrophy Awareness Day, here's a snapshot of 7 investigational agents currently in the DM1 pipeline (and, ...

Michela Leocadi, PhD, research fellow at UCL Queen Square Institute of Neurology, discussed new longitudinal data showing early functional connectivity changes in HD gene carriers years before ...

Twelve observational studies (1997–2025) demonstrated a bidirectional migraine–colic association, including both maternal ...