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Risdiplam (spinal muscular atrophy, < 2 months) ' Benefit assessment according to '35a Social Code Book V 1 Translation of Sections I 1 to I 6 of the dossier assessment Risdiplam (spinale Muskelatrophie, < 2 Monate) – Nutzenbewertung gemäß § 35a SGB V. Please note: This document was translated by an external translator and is provided as a service by IQWiG to English-language readers. However , solely the German original text is absolutely authoritative and legally binding. Risdiplam (spinal muscular atrophy, < 2 months) Benefit assessment according to §35a SGB V1 EXTRACT Project: A23-93 Version: 1.0 Status: 12 December 2023 Extract of dossier assessment A23-93 Version 1.0 Risdiplam ( spinal muscular atrophy, < 2 months) 12 December 2023 Institute for Quality and Efficiency in Health Care
Advice - Extension of reimbursement conditions for risdiplam (Evrysdi) Go to contentYou are here:HomePublicationsAdvice - Extension of reimbursement conditions for risdiplam (Evrysdi®)Search within English part of National Health Care InstituteOpen search boxAdvice - Extension of reimbursement conditions for risdiplam (Evrysdi®)The National Health Care Institute has advised the Minister of Health , Welfare and Sport (VWS) to also reimburse risdiplam (Evrysdi®) from the basic health insurance package for children aged between 0 and 2 months. This medicinal product can be used for children and young adults with the muscular disease SMA. Risdiplam is already included in the Medicines Reimbursement System (GVS) with additional conditions for reimbursement. If the Minister adopts our advice
Nusinersen and risdiplam for treating spinal muscular atrophy Published: 4 Jun 2024 Driving Better Decision-Making in Healthcare Page 1 Oo Nusinersen and risdiplam for treating spinal muscular atrophy Technology Guidance from the MOH Drug Advisory Committee Guidance Recommendations The Ministry of Health’s Drug Advisory Committee has recommended: ✓ Risdiplam 0.75 mg/mL powder for oral solution for treating: ▪ Symptomatic Type 1, 2 or 3 spinal muscular atrophy (SMA) in patients who are/were 18 years of age or under at the time of initial treatment with risdiplam or nusinersen; ▪ Symptomatic Type 1, 2 or 3 SMA in patients aged 19 years or above who had not initiated treatment with risdiplam or nusinersen prior to 19 years of age despite onset of signs/symptoms of SMA; ▪ Pre-symptomatic SMA
SMA-Therapies: Mid- to longer-term follow-up of spinal muscular atrophy (SMA) patients treated for ?24 months with nusinersen or onasemnogene abeparvovec as monotherapies or in combination, and patients treated for ?12 months with risdiplam as monotherap ENGLISH | DEUTSCH ATOM RSS 1.0 RSS 2.0SIMPLE SEARCHADVANCED SEARCHHELPSERVICESLOGINBrowseTypeSubjectAuthor / EditorInstitutionYear AIHTA - Publications - Search - SMA-Therapies: Mid- to longer-term follow-up of spinal muscular atrophy (SMA) patients treated for ≥24 months with nusinersen or onasemnogene abeparvovec as monotherapies or in combination, and patients treated for ≥12 months with risdiplam as monotherapy or in combinationGiess, D. and Erdos, J. and Wild, C. (2023): SMA-Therapies: Mid- to longer-term follow-up of spinal muscular
Risdiplam (Evrysdi) in spinal muscular atrophy. Continue the evaluation Register online | Log in | My PrescrireISSUE CONTENTSTOPICSABOUT PRESCRIREOFFERSenglish.prescrire.org > Spotlight > 100 most recent > Risdiplam (Evrysdi°) in spinal muscular atrophy. Continue the evaluationSpotlightEvery month, the subjects in Prescrire’s Spotlight.100 most recent : 1 | 10 | 20 | 30 | 40 | 50 | 60 | 70 | 80 In infants with symptomatic type 1 spinal muscular atrophy, risdiplam appears to prolong survival, reduce respiratory support requirements and improve certain motor functions, when compared with "historical controls". However, marked disability remained, and the drug’s effect beyond 3 years is unknown. Risdiplam is of dubious efficacy in types 2 and 3 spinal muscular atrophy. Its main adverse effects
Risdiplam (spinal muscular atrophy) - Benefit assessment according to §35a Social Code Book V 1 Translation of Sections 2.1 to 2.7 of the dossier assessment Risdiplam (spinale Muskelatrophie) – Nutzenbewertung gemäß § 35a SGB V (Version 1.0; Status: 29 July 2021). Please note: This translation is provided as a service by IQWiG to English -language readers. However, solely the German original text is absolutely authoritative and legally binding. Extract IQWiG Reports – Commission No. A21-50 Risdiplam (spinal muscular atrophy) – Benefit assessment according to §35a Social Code Book V1 Extract of dossier assessment A21-50 Version 1.0 Risdiplam (spinal muscular atrophy) 29 July 2021 Institute for Quality and Efficiency in Health Care (IQWiG
Risdiplam (spinal muscular atrophy) - Addendum to Commission A21-50 1 Translation of addendum A21-118 Risdiplam (spinale Muskelatrophie) – Addendum zum Auftrag A21-50 (Version 1.0; Status: 1 October 2021). Please note: This translation is provided as a service by IQWiG to English-language readers. However, solely the German original text is absolutely authoritative and legally binding. Addendum 1 October 2021 1.0 Commission: A21-118 Version: Status: IQWiG Reports – Commission No. A21-118 Risdiplam (spinal muscular atrophy) – Addendum to Commission A21-501 Addendum A21-118 Version 1.0 Risdiplam – Addendum to Commission A21-50 1 October 2021 Institute for Quality and Efficiency in Health Care (IQWiG) - i - Publishing details Publisher Institute
Risdiplam (Evrysdi) Terms of use - Canada.ca * Skip to main content * Skip to "About government" Language selection * FrançaisSearchSearch Canada.ca Search Topics menuMain Menu You are here: 1. Home 2. Health 3. Drug and health products 4. Licensing, authorizing and manufacturing drug and health products 5. Drug and health product review and approval 6. Clinical information on drugs
Risdiplam (Evrysdi) - for the treatment of 5q spinal muscular atrophy (SMA) 1 Published 07 February 2022 1 SMC2401 risdiplam 0.75mg/mL powder for oral solution (Evrysdi®) Roche Products Limited 14 January 2021 The Scottish Medicines Consortium (SMC) has completed its assessment of the above product and advises NHS Boards and Area Drug and Therapeutic Committees (ADTCs) on its use in NHSScotland. The advice is summarised as follows: ADVICE: following a full submission assessed under the orphan process risdiplam (Evrysdi®) is accepted for use within NHSScotland. Indication under review: for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA type 1, type 2 or type 3 or with one to four SMN2 [survival of motor neuron 2
Risdiplam (Evrysdi) - To treat spinal muscular atrophy Drug Approval Package: EVRYSDI * Skip to main page content * Skip to search * Skip to topics menu * Skip to common linksHHS U.S. Department of Health and Human Services U.S. Food and Drug Administration * Follow FDA * En EspañolSearch FDASubmit search * Popular Content * Home * Food * Drugs * Medical Devices * Radiation-Emitting Products
Exploration of adverse events associated with risdiplam use: Retrospective cases from the US Food and Drug Administration Adverse Event Reporting System (FAERS) database. Risdiplam is a new drug for treating spinal muscular atrophy (SMA). However, pharmacovigilance analyses are necessary to objectively evaluate its safety-a crucial step in preventing severe adverse events (AEs). Accordingly , the primary objective of the current study was to examine the AEs associated with risdiplam use based on real-world data obtained from the US Food and Drug Administration Adverse Event Reporting System (FAERS) database. More specifically, we examined incidents reported between the third quarter of 2020 and the second quarter of 2023. The imbalance of risdiplam-related AEs was evaluated by computing
Risdiplam therapy in adults with 5q-SMA: observational study on motor function and treatment satisfaction. We aimed to describe the experience of a single neuromuscular center in Germany in treating adult spinal muscular atrophy (SMA) patients with risdiplam and to analyze motor function and treatment satisfaction during a follow-up period up to 20 months. Fourteen patients with type 2 or 3 SMA meaningful improvement (53.8%) and six showed clinically meaningful deterioration (46.2%). There was no treatment withdrawal during the follow-up. The most common adverse events were skin rash/increased skin sensitivity to sunlight (n = 3), diarrhea (n = 3), aphthous ulcer (n = 3) and abdominal pain (n = 2). Most patients stated to be at least "satisfied" with the medication. Risdiplam was well tolerated
Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA). 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 efficacy and safety of risdiplam in type 2 and non‑ambulant type 3 SMA. The primary endpoint was met: a significantly greater change from baseline in 32-item Motor Function Measure (MFM32) total score was observed with risdiplam compared with placebo at month 12. After 12 months, all participants received risdiplam while preserving initial treatment blinding. We report 24-month efficacy and safety results in this population
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial Risdiplam is an oral small molecule approved for the treatment of patients with spinal muscular atrophy, with approval for use in patients with type 2 and type 3 spinal muscular atrophy granted on the basis of unpublished data. The drug modifies pre-mRNA splicing of the SMN2 gene to increase production of functional SMN. We aimed to investigate the safety and efficacy of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy. In this phase 3, randomised, double-blind, placebo-controlled study, patients aged 2-25 years with confirmed 5q autosomal recessive type 2 or type 3 spinal muscular
Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment. Spinal muscular atrophy (SMA) is caused by reduced levels of survival of motor neuron (SMN) protein due to deletions and/or mutations in the SMN1 gene. Risdiplam is an orally administered molecule that modifies SMN2 pre-mRNA splicing to increase functional SMN protein. SUNFISH Part 1 was a dose-finding study conducted in 51 individuals with types 2 and 3 SMA aged 2-25 years. A dose-escalation method was used to identify the appropriate dose for the subsequent pivotal Part 2. Individuals were randomized (2:1) to risdiplam or placebo at escalating dose levels for a minimum 12-week, double-blind, placebo-controlled period, followed by treatment for 24
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial. Risdiplam is an orally administered therapy that modifies pre-mRNA splicing of the survival of motor neuron 2 (SMN2) gene and is approved for the treatment of spinal muscular atrophy. The FIREFISH study is investigating the safety and efficacy of risdiplam in treated infants with type 1 spinal muscular atrophy versus historical controls. The primary endpoint of part 2 of the FIREFISH study showed that infants with type 1 spinal muscular atrophy attained the ability to sit without support for at least 5 s after 12 months of treatment. Here, we report on the safety and efficacy of risdiplam in FIREFISH part 2 over 24 months of treatment. FIREFISH
A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier. Risdiplam (RG7916, RO7034067) is an orally administered, centrally and peripherally distributed, survival of motor neuron 2 (SMN2) mRNA splicing modifier for the treatment of spinal muscular atrophy (SMA). The objectives of this entry-into-human study were to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of risdiplam, and the effect of the strong CYP3A inhibitor itraconazole on the PK of risdiplam in healthy male volunteers. Part 1 had a randomized, double-blind, adaptive design with 25 subjects receiving single ascending oral doses of risdiplam (ranging from 0.6-18.0 mg, n = 18) or placebo (n