Results 61 to 70 of about 45,438 (256)
Perancangan Kampanye Interaktif tentang Pendeteksian Dini Spinal Muscular Atrophy [PDF]
, 2023 Walaupun diperkirakan 1 dari 6.000 sampai 10.000 bayi yang lahir memiliki penyakit spinal muscular atrophy, masih belum adanya laporan resmi mengenai jumlah penderita spinal muscular atrophy di Indonesia.
Elizabeth, Graciella
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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 Genotype Distribution.
, 2013 Spinal Muscular Atrophy Genotype Distribution.
Chih-Chao Yang (160072) +10 more
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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
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
Spinal muscular atrophy (Werdnig‑Hoffmann atrophy disease)
, 2018 Introduction. Spinal muscular atrophy type 1 is an autosomal recessive neuromuscular disorder characterized by degeneration of the anterior horn cells in the spinal cord, leading to symmetric muscle weakness and atrophy.
Mariana A. RYZNYCHUK +4 more
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, 1990 Juvenile chronic segmental spinal muscular atrophy of Hirayama is described in two adult identical twins from the Department of Neurology, University of Vermont College of Medicine, Burlington ...
J Gordon Millichap
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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
Severe Spinal Muscular Atrophy Variant Associated With Congenital Bone Fractures
, 2016 Infantile autosomal recessive spinal muscular atrophy (type I) represents a lethal disorder leading to progressive symmetric muscular atrophy of limb and trunk muscles. Ninety-six percent cases of spinal muscular atrophy type I are caused by deletions or
Bührer, Christoph +6 more
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