The approval, based on phase 3 IB1001-303 trial data showing consistent SARA scale improvement, makes levacetylleucine the first treatment approved specifically for ataxia-telangiectasia.
Ulefnersen met its primary end point in the phase 3 FUSION trial, providing the first placebo-controlled evidence for a genetically targeted therapy in FUS-ALS.
A recently published study showed that Qihuang needle acupuncture significantly improved motor function, nonmotor symptoms, and quality of life in patients with PD when added to standard medication.
The CHMP recommended approval of gefurulimab (Klygefa), a weekly, self-administered C5 inhibitor, for AChR-antibody-positive generalized myasthenia gravis in the EU.
Brian Lin, PhD, discussed the current evidence linking football participation with ALS, the questions that remain unanswered, and how emerging research may help clarify the association.
Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Jennifer Fokas, MD, and Minali Nigam, MD. [LISTEN TIME: 21 minutes]