Results 131 to 140 of about 1,977 (181)

Fibro-adipogenic progenitor cells from murine SMA muscles are intrinsically adipogenic. [PDF]

Proc Natl Acad Sci U S A
Luo YE, Abe-Teh Z, Alsaghir T, Heiden KD, Basso KB, Barton ER.   +5 more
europepmc   +1 more source

Preventing spinal muscular atrophy through the national premarital screening program in Türkiye: an economic comparison with treatment costs. [PDF]

Turk J Med Sci
Dur G, Tecirli G, Okumuş N.
europepmc   +1 more source

MyomiR Networks in Spinal Muscular Atrophy: Associations With Clinical Severity and Treatment Response. [PDF]

Mol Neurobiol
Barbo M, Koritnik B, Leonardis L, Dolžan V, Ravnik-Glavač M.   +4 more
europepmc   +1 more source

Elective preterm birth for earlier spinal muscular atrophy treatment. [PDF]

Mol Ther Adv
Dangouloff T, Chantraine F, Hennuy N, Rigo V, Tribolet S, Brande LV, Servais L.   +6 more
europepmc   +1 more source

Patients on treatment with risdiplam in Italy: challenges in the interpretation of the real-world data

Neurological Sciences
(i) provide a snapshot from a large cohort of Italian patients with SMA on risdiplam in the real-world setting; (ii) identify any differences in the cohorts before and after commercial drug approval considering the different eligibility access criteria (iii) describe preliminary data on adherence to treatment and reasons for shifting from nusinersen to
Claudia Dosi, Eugenio Mercuri, Pini Antonella   +2 more
exaly   +11 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 ...
Julia Paik (6912664)
openaire   +3 more sources

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Combination therapy with onasemnogene and risdiplam in spinal muscular atrophy type 1

Muscle and Nerve, 2021
AbstractIntroduction/AimsThere are currently three medications approved for spinal muscular atrophy (SMA), but the use of these medications in combination has not been well described.MethodsThis is a retrospective report of four cases of SMA treated with dual onasemnogene and risdiplam therapy at our institution.ResultsFollowing onasemnogene therapy ...
Michael Cartwright
exaly   +3 more sources

Short Review on Currently Used Sample Preparation and Determination Methods of Risdiplam

Journal of Separation Science
ABSTRACT Risdiplam is a new therapeutic agent developed to treat spinal muscular atrophy (SMA), a genetic neurodegenerative disease caused by mutations in the SMN1 gene. Unlike previous invasive therapies, risdiplam offers the advantage of oral administration, significantly ...
Balińska, Natalia, Ostrowska, Karolina, Studzińska, Sylwia   +2 more
exaly   +4 more sources

Transitioning From Nusinersen to Risdiplam for Spinal Muscular Atrophy in Clinical Practice: A Single‐Center Experience

Muscle and Nerve
BackgroundNusinersen and risdiplam are U.S. Food and Drug Administration (FDA)-approved treatments for spinal muscular atrophy (SMA). No head-to-head clinical trials to assess efficacy exist. Observational studies are needed to determine if transitioning
Megan Waldrop, Kavitha Kotha, Natalie Reash   +2 more
exaly   +2 more sources

Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA) [PDF]

Journal of Neurology, 2023
Risdiplam is an oral, survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier approved for the treatment of spinal muscular atrophy (SMA). SUNFISH (NCT02908685) Part 2, a Phase 3, randomized, double-blind, placebo-controlled study, investigated the ...
Heidemarie Kletzl, Laurent Servais, John W Day   +2 more
exaly   +4 more sources