Results 71 to 80 of about 2,700 (197)
British Journal of Clinical Pharmacology, EarlyView.With multiple disease‐modifying therapies now available, treatment switching has become an important clinical consideration in the management of spinal muscular atrophy (SMA). While some switches are prompted by suboptimal clinical response, more commonly they are driven by treatment burden, convenience, or adverse events.
Andrej Belančić +4 more
wiley +1 more source
British Journal of Clinical Pharmacology, EarlyView.Small and mid‐sized pharmaceutical innovators often have limited in‐house health economics and market access expertise, and may struggle to align development strategies of investigational medicinal products with health system needs and payer expectations.
Zoltán Kaló +5 more
wiley +1 more source
British Journal of Clinical Pharmacology, EarlyView.Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić +4 more
wiley +1 more source
Molecular Therapy: Methods & Clinical DevelopmentSpinal muscular atrophy is a neurodegenerative disorder resulting from the irreversible loss of anterior horn cells secondary to homozygous mutations in the survival motor neuron gene SMN1.
Cassie Ables +6 more
doaj +1 more source
How the HTAR will contribute to a value‐based decision‐making for medicinal products across the EU
British Journal of Clinical Pharmacology, EarlyView.The European Union Health Technology Regulation 2021/2282 (HTAR) introduces joint assessment of health technologies (including medicinal products and medical devices) across EU Member States. It was signed into law in 2021 and came into full force in January 2025.
Roisin Adams, Michal Stanak
wiley +1 more source
British Journal of Clinical Pharmacology, EarlyView.Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić +7 more
wiley +1 more source
Cost-Effectiveness Analysis of Newborn Screening for Spinal Muscular Atrophy in Italy. [PDF]
Background and Objective Untreated spinal muscular atrophy (SMA) is the leading genetic cause of death in children younger than 2 years of age. Early detection through newborn screening allows for presymptomatic diagnosis and treatment of SMA.
Bischof M +5 more
core +1 more source
Muscle &Nerve, EarlyView.ABSTRACT Introduction/Aims Three disease‐modifying therapies are approved for individuals with spinal muscular atrophy (SMA); however, data concerning the combination of these therapies remain limited. This study aimed to evaluate the safety and efficacy of add‐on risdiplam in children who had experienced clinical deterioration despite gene therapy ...
Corinna Stoltenburg +4 more
wiley +1 more source
Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec [PDF]
Onasemnogene abeparvovec; Spinal muscular atrophy; Survival motor neuron 2 geneOnasemnogene abeparvovec; Atròfia muscular espinal; Gen de la neurona motora 2 de supervivènciaOnasemnogene abeparvovec; Atrofia muscular espinal; Gen de la neurona motora 2 ...
Aharoni, Sharon +5 more
core +1 more source
Onasemnogene abeparvovec for spinal muscular atrophy: The costlier drug ever
International Journal of Applied and Basic Medical Research, 2019 Rajiv Mahajan
openalex +5 more sources