Results 11 to 20 of about 1,718 (175)

Short-term safety results from compassionate use of risdiplam in patients with spinal muscular atrophy in Germany

Orphanet Journal of Rare Diseases, 2022
Background The oral, selective SMN2-splicing modifier risdiplam obtained European approval in March 2021 for the treatment of patients ≥ 2 months old with a clinical diagnosis of 5q-associated spinal muscular atrophy (SMA) 1/2/3 or with 1–4 SMN2 gene ...
Andreas Hahn, René Günther, Albert Ludolph, Oliver Schwartz, Regina Trollmann, Patrick Weydt, Markus Weiler, Kathrin Neuland, Martin Sebastian Schwaderer, Tim Hagenacker, the Risdiplam Compassionate Use Program Group   +10 more
doaj   +3 more sources

Risdiplam for the Use of Spinal Muscular Atrophy [PDF]

Orthopedic Reviews, 2021
Spinal muscular atrophy (SMA) is one of the leading causes of death in infants related to the degeneration of neurons. Currently, there are no curative treatment options for SMA, and many options available may not be feasible. This review presents the background, clinical studies, and indications for the use of Risdiplam in treating SMA.
Juyeon, Kakazu, Nakoma L, Walker, Katherine Claire, Babin, Katherine A, Trettin, Christopher, Lee, Patricia B, Sutker, Adam M, Kaye, Alan D, Kaye   +7 more
openaire   +4 more sources

Health economic evaluation of risdiplam in patients with spinal muscular atrophy

Фармакоэкономика, 2021
Background. Spinal muscular atrophies (SMA) are clinically and genetically heterogenous inherited orphan diseases leading to progressive spinal motoneurons degeneration and loss of function.
А. S. Kolbin, А. А. Kurylev, Yu. Е. Balykina, М. А. Proskurin, S. А. Mishinova   +4 more
doaj   +2 more sources

The evolving therapeutic landscape of spinal muscular atrophy - A scoping review of investigational agents, emerging delivery technologies and strategic innovations. [PDF]

Br J Clin Pharmacol
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Belančić A, Eustaquio P, Gkrinia EMM, Stević I, Javor E, Lam YW, Janković S, Vitezić D.   +7 more
europepmc   +2 more sources

Effect of mild or moderate hepatic impairment on the pharmacokinetics of risdiplam

British Journal of Clinical Pharmacology, 2022
AimThis phase I, multicentre, open‐label, nonrandomised, parallel‐group, two‐part study aimed to evaluate the effect of mild to moderate hepatic impairment on the pharmacokinetics (PK), safety and tolerability of a single oral dose of risdiplam.MethodsAdult subjects (aged 18‐70 years) with mild (Child‐Pugh Class A; Part 1) or moderate (Child‐Pugh Class
Heidemarie Kletzl
exaly   +3 more sources

Diverging Safety Signals: A Trend Analysis of Suspected Adverse Drug Reactions Reporting for Spinal Muscular Atrophy Therapies in the European Union [PDF]

Neurology International
Background/Objectives: The approval of disease-modifying therapies has significantly improved outcomes for patients with spinal muscular atrophy (SMA), yet their long-term safety profiles remain under continuous evaluation.
Andrej Belančić, Petar Mas, Ivana Stević, Dinko Vitezić, Slobodan Janković   +4 more
doaj   +2 more sources

Assessment of safety and efficacy of risdiplam treatment in adults with spinal muscular atrophy [PDF]

Frontiers in Neurology
IntroductionRisdiplam has been shown to be safe, well tolerated, and improves or stabilizes motor function in individuals with SMA, but limited published data exists for adults.
Andrea Jaworek, Kathryn Jira, Matti Allen, Songzhu Zhao, Kristina Kelly, Trevor Moravec, Marco Tellez, Sarah Heintzman, Jerold Reynolds, Gary Sterling, Stephen J. Kolb, William David Arnold, Bakri Elsheikh   +12 more
doaj   +2 more sources

Safety of Risdiplam in Japanese Patients with Spinal Muscular Atrophy: A 12‑Month Interim Analysis of a Postmarketing Surveillance Study [PDF]

Neurology and Therapy
Introduction Risdiplam, an oral splicing modifier for the survival motor neuron-2 gene (SMN2), is approved for treating spinal muscular atrophy (SMA).
Kayoko Saito, Toshio Saito, Reiko Arakawa, Yasuhiro Takeshima, Hisahide Nishio, Yuka Ishikawa, Masahisa Katsuno, Takahiko Tsumuraya, Hiromitsu Kawata, Yuki Miyano, Hirofumi Komaki   +10 more
doaj   +2 more sources

Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series [PDF]

BMC Neurology
Background Spinal muscular atrophy (SMA) is caused by deletions or mutations in the survival of motor neuron (SMN) 1 gene resulting in progressive motor function loss, and additional disease-related complications, including dysphagia and respiratory ...
Melissa D. Svoboda, Nancy Kuntz, Carmen Leon-Astudillo, Barry J. Byrne, Jena Krueger, Jennifer M. Kwon, Cory Sieburg, Diana Castro   +7 more
doaj   +2 more sources

Liver Steatosis in Induced Hepatocytes From Carriers of Spinal Muscular Atrophy. [PDF]

Muscle Nerve
ABSTRACT Introduction/Aims Although classically characterized as a motor neuron disease, spinal muscular atrophy (SMA) is increasingly recognized as a multisystem disorder. We previously showed hepatocyte‐intrinsic steatosis in SMA, raising the question of whether SMA carriers, who are typically asymptomatic, may also exhibit subclinical hepatic ...
Sun L, Leow DMK, Wang LC, Eio MY, Koh HWL, Khong ZJ, Narayanan G, Teo AKS, Giadone R, Sobota RM, Ng SY, Teo AKK, Ong WY, Rubin LL, Darras BT, Yeo CJJ.   +15 more
europepmc   +2 more sources