Results 131 to 140 of about 1,718 (175)

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
Maria Sframeli, Noémi Brolatti, Chiara Bravetti   +2 more
exaly   +9 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

Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA)

Expert Opinion on Investigational Drugs, 2022
peer reviewedINTRODUCTION: Spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disease which is characterised by muscle atrophy and early death in most patients.
Theodora Markati, Gemma Fisher, Sithara Ramdas   +2 more
exaly   +3 more sources

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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 ...
Sylwia Studzinska
exaly   +4 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 ...
Maryam Oskoui, John W Day, Nicolas Deconinck   +2 more
exaly   +3 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 Iammarino, Kavitha Kotha, Natalie F Miller   +2 more
exaly   +2 more sources

Switching from Nusinersen to Risdiplam: A Croatian Real-World Experience on Effectiveness and Safety

Journal of Personalized Medicine
: (1) Background: Data on combination or sequential treatment of spinal muscular atrophy (SMA) with disease-modifying drugs (DMDs) are missing and the latter field is poorly understood.
Andrej Belančić, Marta Kucan, Dinko Vitezic   +2 more
exaly   +2 more sources

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

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

Muscle & 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 ...
Kelly F, Oechsel, Michael S, Cartwright
openaire   +2 more sources

JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam [PDF]

Journal of Neurology
Risdiplam is a once-daily oral, survival of motor neuron 2 (SMN2) splicing modifier approved for the treatment of spinal muscular atrophy (SMA). JEWELFISH (NCT03032172) investigated the safety, tolerability, pharmacokinetics (PK), and PK/pharmacodynamic (
Francesco Muntoni, Paulo Fontoura, Renata S Scalco   +2 more
exaly   +1 more source