Results 161 to 170 of about 121,613 (384)

PREGNANCY AND CHILDBIRTH IN A PATIENT WITH SPINAL MUSCULAR ATROPFY (CLINICAL CASE)

open access: yesМать и дитя в Кузбассе, 2020
Spinal muscle atrophy (SMA) is a group of diseases inherited by an autosomal recessive type and characterized by degeneration of cells of the anterior horns of the spinal cord.
Ирина Анатольевна Ушакова   +6 more
doaj  

Air stacking: effects on pulmonary function in patients with spinal muscular atrophy and in patients with congenital muscular dystrophy, [PDF]

open access: diamond, 2014
Tanyse Bahia Carvalho Marques   +5 more
openalex   +1 more source

Single‐Dose Gene‐Replacement Therapy for Spinal Muscular Atrophy

open access: yesNew England Journal of Medicine, 2017
J. Mendell   +25 more
semanticscholar   +1 more source

Switching disease‐modifying therapies in patients with spinal muscular atrophy: A systematic review on effectiveness outcomes

open access: yesBritish 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

Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait

open access: yesNeurology International
Spinal muscular atrophy is a neuromuscular genetic condition associated with progressive muscle weakness and atrophy. Nusinersen is an antisense oligonucleotide therapy approved for the treatment of 5q spinal muscular atrophy in pediatric and adult ...
Asma AlTawari   +12 more
doaj   +1 more source

Nusinersen versus Sham Control in Infantile‐Onset Spinal Muscular Atrophy

open access: yesNew England Journal of Medicine, 2017
R. Finkel   +397 more
semanticscholar   +1 more source

Early‐stage health technology assessment of a curative gene therapy for multiple sclerosis

open access: yesBritish 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

Considerations for Treatment in Clinical Care of Spinal Muscular Atrophy Patients

open access: yesChildren
Spinal 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

Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy [PDF]

open access: gold, 2017
Kevin A. Kaifer   +6 more
openalex   +1 more source

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