Results 111 to 120 of about 605,611 (288)
Early‐stage health technology assessment of a curative gene therapy for multiple sclerosis
British Journal of Clinical Pharmacology, EarlyView.Aims Multiple sclerosis (MS) is associated with significant early morbidity, reduced life expectancy and substantial healthcare and societal costs. The primary objective of this study is to assess the early cost‐effectiveness potential of a novel gene therapy, IMMUTOL, for MS compared with current high‐efficacy treatment sequences.
Attila Imre, Balázs Nagy, Rok Hren
wiley +1 more source
Spinal Muscular Atrophy Type III
, 2011 Spinal muscular atrophy is a relatively stable chronic disease. Patients may gradually experience declines in muscle strength and motor function over time.
Jacqueline Montes +5 more
core +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
Considerations for Treatment in Clinical Care of Spinal Muscular Atrophy Patients
ChildrenSpinal Muscular Atrophy is a neurodegenerative disease which can lead to muscle weakness, paralysis, and in some cases death. There are many factors that contribute to the severity of symptoms and those factors can be used to determine the best course of
Stephanie Voight, Kapil Arya
doaj +1 more source
, 2022 Lo Spinal Muscular Atrophy Lab è il primo “laboratorio” di management che ha l’obiettivo di supportare i sistemi sanitari (regionali e aziendali), i professionisti e i pazienti nel migliorare la qualità delle cure per le persone affette da ...
Tozzi, Valeria D. +2 more
core
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
BMC AnesthesiologyBackground Spinal Muscular Atrophy (SMA) is a rare autosomal recessive genetic disorder characterized by degeneration of motor neurons in the spinal cord, resulting in progressive limb muscle weakness, atrophy, and severe scoliosis.
Zhuangyuan Chen +5 more
doaj +1 more source
The effects of fluphenazine on the neuromuscular phenotype in a mouse model of spinal muscular atrophy [PDF]
Spinal muscular atrophy (SMA) is a degenerative neuromuscular disorder caused by a mutation in the survival motor neuron 1 gene (SMN1), which renders the gene dysfunctional. SMN protein is vital for the survival of motor neurons.
Haynes, Katie Louise
core
Precision medicine in paediatrics: Progress and priorities
British Journal of Clinical Pharmacology, EarlyView.Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain +3 more
wiley +1 more source
Objectives This study aims to systematically collect data on cost-effectiveness analyses that assess technologies to treat type I and II spinal muscular atrophy and evaluate their recommendations.
Carvalho, Lélia +5 more
core +1 more source