The growing number of people living with Parkinson’s disease (PD), multiple sclerosis (MS) and motor neuron disease (MND) is being driven by different underlying factors, according to new research due to be presented at the European Academy of Neurology (EAN) Congress 2026 and published in Neurology [1,2]. Researchers from France and Sweden analysed nationwide health…
Category: Neuromuscular
NICE recommends givinostat for Duchenne muscular dystrophy
The National Institute for Health and Care Excellence has recommended Givinostat for use on the NHS in England as a treatment option for some people with Duchenne muscular dystrophy, marking another milestone for patients living with the rare inherited condition. Under final draft guidance, NICE said the treatment can be offered to patients aged six…
Roche launches new Elevidys trial to address EU rejection in Duchenne therapy bid
Roche is initiating a new global phase 3 clinical trial of its Duchenne muscular dystrophy gene therapy, Elevidys, following a previous rejection from European regulators, as the company seeks to strengthen evidence for approval in ambulatory patients. The Swiss pharmaceutical group is aiming to address concerns raised by the European Medicines Agency, which last year…
FDA approves higher dose nusinersen for spinal muscular atrophy
The US Food and Drug Administration has approved a higher dose regimen of nusinersen for the treatment of spinal muscular atrophy, marking a significant development nearly a decade after the therapy was first introduced. The updated dosing approach increases the concentration of nusinersen administered during both the initial loading phase and ongoing maintenance treatment. It…
Scotland pilots newborn screening for spinal muscular atrophy
Scotland has become the first nation in the UK to introduce routine newborn screening for spinal muscular atrophy, marking a significant step forward in early diagnosis and treatment of the rare genetic condition. The screening will be delivered through the existing heel prick blood spot test offered to all newborns, as part of a two…
FDA approves Uplizna for adults with generalised myasthenia gravis
Amgen says the US Food and Drug Administration has approved Uplizna (inebilizumab-cdon) to treat adults with generalised myasthenia gravis (gMG) who are positive for anti-acetylcholine receptor (AChR) or anti-muscle specific tyrosine kinase (MuSK) antibodies, adding a new targeted option for a condition marked by fluctuating muscle weakness that can affect breathing, swallowing, speech and vision….
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…
Pyrimidine Nucleos(t)ide Therapy Shows 95% Reduction in Risk of Death in TK2 Deficiency
Results published in Neurology have highlighted a 95% reduction in the risk of death among patients treated with pyrimidine nucleoside and/or nucleotide therapy for thymidine kinase 2 deficiency (TK2d), a rare mitochondrial disorder. The findings, from a multicentre retrospective chart review, represent the first published data exploring the safety and efficacy of this investigational therapy…
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…
