Children, adolescents see Spinraza benefits in real-world study Written by Andrea Lobo | Oct. 26, 2026 Treatment with Spinraza (nusinersen) led to sustained improvements in motor function in children and adolescents with spinal muscular atrophy (SMA), according to a real-world study from Czechia…
In amyotrophic lateral sclerosis (ALS), motor neurons in the spinal cord and central nervous system die off for unknown reasons, causing creeping paralysis and, eventually, death. Mutations in the... In amyotrophic lateral sclerosis (ALS), motor neurons in the spinal cord…
A new review argues that disease-specific HDAC isoforms, not broad-spectrum inhibition, should guide epigenetic drug development for neurodegenerative diseases.
Alnylam Pharmaceuticals, Inc. (ALNY) RNAi Roundtable: Exon 1 Targeting With Brezosiran for Huntington's Disease October 26, 2026 10:00 AM EDTCompany... Alnylam Pharmaceuticals, Inc. (ALNY) RNAi Roundtable: Exon 1 Targeting With Brezosiran for Huntington's Disease October 26, 2026 10:00 AM EDT Joshua Brodsky - Vice President of Investor Relations Hello,…
Researcher awarded $300,000 FightMND grant to study fast motor neurons, the most vulnerable cells in neurodegenerative disease’s progression Fast motor neurons are the harbinger of Motor Neurone Disease (MND), the first domino in the nerve cell system to fall in people…
For a Butler County couple whose plans for traveling were dashed, or a woman from Bellevue who took a chance at having a daughter, just knowing they are not alone means so much. But for those living with facioscapulohumeral muscular…
Researcher awarded $300,000 FightMND grant to study fast motor neurons, the most vulnerable cells in neurodegenerative disease's progression Fast motor neurons are the harbinger of Motor Neurone Disease (MND), the first domino in the nerve cell system to fall in people…
Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients aged 2 and older without early treatment. FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration challenges, with key…
A machine learning tool combining quantitative muscle MRI fat fractions with clinical scores predicts when boys with Duchenne muscular dystrophy will lose ambulation, achieving strong accuracy up to four years ahead and stratifying patients into risk groups with starkly different…
Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered intrathecally quarterly, both showing similar safety and efficacy profiles. Clinical Brief: Disease-Modifying Therapies for…
ALS, Huntington's disease, spinal muscular atrophy, prion disease and other rare neurological disorders: their biology and new treatments.
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