Results 141 to 150 of about 2,038 (184)

Course of joint range of motion in children with spinal muscular atrophy receiving disease-modifying treatment. [PDF]

Orphanet J Rare Dis
Oude Lansink ILB, Gorter JW, van der Pol WL, Vd Woude DR, Scheiberlich PJ, van Eijk RPA, Bartels B, Beelen A.   +7 more
europepmc   +1 more source

Risdiplam in Adult Patients With 5q Spinal Muscular Atrophy: A Single-Center Longitudinal Study. [PDF]

Muscle Nerve
Gavriilaki M, Moschou M, Pagiantza M, Arnaoutoglou M, Kimiskidis V.   +4 more
europepmc   +1 more source

Spinal muscular atrophy in the era of newborn screening: how the classification could change. [PDF]

Front Neurol
Varone A, Esposito G, Bitetti I.
europepmc   +1 more source

The Effect of Long-Term Non-Invasive Ventilation on Tracheostomy-Free Survival and Hospitalizations in Types 2 and 3 Spinal Muscular Atrophy Patients. [PDF]

J Clin Med
Vianello A, Guarnieri G, Bertagna De Marchi L, Molena B, Arcaro G, Capece G, Sogus E, Lionello F, Pegoraro E.   +8 more
europepmc   +1 more source

Prenatal Management of Spinal Muscular Atrophy in the Era of Genetic Screening and Emerging Opportunities in In Utero Therapy. [PDF]

Biomedicines
Mežnarić S, Belančić A, Rački V, Vitezić D, Mršić-Pelčić J, Pilipović K.   +5 more
europepmc   +1 more source

Newborn screening for spinal muscular atrophy in Ukraine: from pilot project to national programme. [PDF]

Lancet Reg Health Eur
Olkhovych N, Samonenko N, Shkliarska T, Hill S, Grechanina O, Veropotvelyan M, Makukh H, Barvinska O, Mytsyk N, Dangouloff T, Ivanova T, Servais L, Gorovenko N.   +12 more
europepmc   +1 more source

Risdiplam for spinal muscular atrophy.

Australian prescriber
openaire   +1 more source

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Risdiplam: First Approval

Drugs, 2020
Risdiplam (Evrysdi™) is an orally administered, survival motor neuron 2 (SMN2)-directed RNA splicing modifier being developed by Roche, PTC Therapeutics Inc and the SMA Foundation for the treatment of the spinal muscular atrophy. The small molecule is designed to treat spinal muscular atrophy caused by mutations in chromosome 5q leading to SMN protein ...
openaire   +2 more sources

Risdiplam: A Review in Spinal Muscular Atrophy

CNS Drugs, 2022
Risdiplam (Evrysdi®) is the first oral drug developed to treat spinal muscular atrophy (SMA) and is approved in multiple countries worldwide. It is approved for the treatment of SMA in patients aged ≥ 2 months in the USA and the EU, with this approval further specified in the EU for the treatment of 5q-autosomal recessive SMA with a clinical diagnosis ...
openaire   +2 more sources