The Scottish Medicines Consortium has accepted nusinersen for restricted use within NHS Scotland for babies with genetically confirmed 5q spinal muscular atrophy before symptoms appear. The decision, published in August 2026, provides clinicians with an additional early treatment option for infants identified through newborn screening or targeted genetic testing. The SMC advice confirms that nusinersen…
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Teva files FDA application for ecopipam in paediatric Tourette syndrome
Teva Pharmaceutical Industries has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for ecopipam, an investigational treatment for paediatric Tourette syndrome. The company says the submission marks a significant milestone for what could become the first new FDA-approved therapy for children and adolescents with Tourette syndrome in more than…
FDA fast track for cladribine capsules in gMG
Merck has announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to cladribine capsules for the treatment of generalised Myasthenia Gravis (gMG). The therapy, already recognised with Orphan Drug Designation, is being developed as a potential first oral treatment option for people living with gMG. Cladribine capsules are currently being…
Immediate motor gains in tremor-dominant Parkinson’s disease
MR-guided ultrasound targets PTT circuit A new study in the European Journal of Neurology highlights the immediate therapeutic impact of stepwise dual-target MR-guided focused ultrasound (dtMRgFUS) in treating tremor-dominant Parkinson’s disease. This approach targets both the ventral intermediate nucleus (VIM) and the pallidothalamic tract (PTT) in a single session. Twenty patients (mean age 69.3 ± 8.9 years)…
Eisai presents new clinical data for four years of continuous treatment with lecanemab at Alzheimer’s Association International Conference 2025
Eisai has presented new clinical data demonstrating that early Alzheimer’s disease (AD) patients continue to benefit from four years of Leqembi®▼(lecanemab) treatment, at the Alzheimer’s Association International Conference (AAIC) 2025, held in Toronto, Canada, and virtually on 30 July 2025 [1]. Key takeaways included: Lecanemab is an amyloid-beta (Aβ) monoclonal antibody that preferentially binds and clears…
US shipments of Elevidys paused
Duchenne Muscular Dystrophy gene therapy distribution suspended Sarepta has voluntarily paused all shipments of its Duchenne muscular dystrophy gene therapy Elevidys in the U.S. The FDA made the distribution suspension request Friday after two patient deaths following treatment with Elevidys. More recently, Sarepta disclosed the death of a third patient who was given one of its gene…
Mary Reilly receives AANEM’s 2025 Honorary Member Award
The American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) has announced that Dr Mary Reilly, MD has received the 2025 Honorary Member Award for her scientific contributions and unwavering commitment to improving patient care in neuromuscular medicine. Dr Reilly graduated from University College Dublin in 1986, earned her Doctor of Medicine in 1996, and was…
SMC rejects lecanemab fast track resubmission
The Scottish Medicines Consortium (SMC) has announced that following a Fast Track Resubmission by Eisai, Leqembi® (lecanemab) has not been recommended for use in the National Health Service (NHS) in Scotland1 7 July 2025 – Eisai Europe Ltd. and Biogen Idec Ltd. announced today that the Scottish Medicines Consortium (SMC) has not recommended Leqembi® (lecanemab) for use…
Teva leads call to secure Europe’s access to critical medicines and protect health sovereignty
Speaking at the Medicines for Europe Annual Conference, Michal Nitka, SVP Head Generics Europe & Global Head OTC at Teva Pharmaceuticals, highlights the essential role of a resilient, competitive, and environmentally sustainable pharmaceutical industry in safeguarding patient access to medicines and strengthening Europe’s healthcare sovereignty. While advancing its innovative portfolio of medicines, Teva strongly believes in…
Movement disorders abstract rated top at AAN
Encora breakthrough pivotal data demonstrating superiority over sham recognised as top rated Movement Disorders abstract Encora Therapeutics’ AAN abstract Safety and Efficacy of a Wrist-worn Vibratory Stimulation Wearable for Upper Limb Tremor Reduction in Essential Tremor (ULTRE): A Randomized, Controlled Trial, was honoured as an “Abstract of Distinction” by the American Academy of Neurology (AAN) and the…
