Risdiplam (spinalmuscularatrophy, < 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 (spinalmuscularatrophy, < 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 ( spinalmuscularatrophy, < 2 months) 12 December 2023 Institute for Quality and Efficiency in Health Care
Technology Briefings OAV101 IT for treating spinalmuscularatrophy in children aged 2 and older 28 JANUARY 2025 OAV101 IT for treating spinalmuscularatrophy in children aged 2 and older OAV101 IT is an advanced therapy medicinal product (gene therapy product) in clinical development for the treatment of spinalmuscularatrophy (SMA). SMA is a rare genetic condition that affects the motor
Carrier Screening Programs for Cystic Fibrosis, Fragile X Syndrome, Hemoglobinopathies and Thalassemia, and SpinalMuscularAtrophy: A Health Technology Assessment Skip to main contentAn official website of the United States government Here's how you know Log inAccess keysNCBI HomepageMyNCBI HomepageMain ContentMain NavigationVolume 23(4); 2023Carrier Screening Programs for Cystic Fibrosis , Fragile X Syndrome, Hemoglobinopathies and Thalassemia, and SpinalMuscularAtrophy: A Health Technology AssessmentOntario Health (Quality)Ont Health Technol Assess Ser. 2023; 23(4): 1–398. Published online 2023 Aug 10.PMCID: PMC10453298Article PubReader PDF–5.2MCiteArticles from Ontario Health Technology Assessment Series are provided here courtesy of Ontario HealthFOLLOW NCBIConnect with NLM National
Onasemnogene abeparvovec for treating spinalmuscularatrophy Overview | Onasemnogene abeparvovec for treating spinalmuscularatrophy | Guidance | NICE You are here: 1. NICE 2. NICE Guidance 3. Conditions and diseases 4. Musculoskeletal conditions 5. Spinal conditions Onasemnogene abeparvovec for treating spinalmuscularatrophy * Highly specialised technologies guidance * Reference abeparvovec (Zolgensma) for treating spinalmuscularatrophy in babies.Is this guidance up to date?Next review:For recommendations 1.1 and 1.2: 2024For recommendation 1.3: More evidence on onasemnogene abeparvovec is being collected via the managed access agreement. After this, NICE will decide whether or not to recommend it for use on the NHS and update the guidance. It will be available through
Nusinersen and risdiplam for treating spinalmuscularatrophy Published: 4 Jun 2024 Driving Better Decision-Making in Healthcare Page 1 Oo Nusinersen and risdiplam for treating spinalmuscularatrophy 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 spinalmuscularatrophy (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
Clinical effectiveness and safety of newborn screening for spinalmuscularatrophy. Systematic review. Efectividad clínica y seguridad del cribado neonatal de la atrofia muscular espinal - Servicio de Evaluación de Tecnologías Sanitarias de Andalucía (AETSA) TwitterRssServicio de Evaluación de Tecnologías Sanitarias de Andalucía (AETSA)La misión del Servicio de Evaluación de Tecnologías Sanitaria * Producción * Trabajos en marcha * Producción científica * Trabajo en red * Red de agencias del SNS (RedETS). * EUnetHTA-JA3 * Euroscan * INAHTA * Actividades * Formación * Colabora con nosotros * Eventos Efectividad clínica y seguridad del cribado neonatal de la atrofia muscular espinalVolver al buscadorClinical effectiveness and safety of newborn screening for spinalmuscularatrophy. Systematic review
SMA-Therapies: Mid- to longer-term follow-up of spinalmuscularatrophy (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 spinalmuscularatrophy (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 spinalmuscular
Risdiplam (Evrysdi) in spinalmuscularatrophy. Continue the evaluation Register online | Log in | My PrescrireISSUE CONTENTSTOPICSABOUT PRESCRIREOFFERSenglish.prescrire.org > Spotlight > 100 most recent > Risdiplam (Evrysdi°) in spinalmuscularatrophy. Continue the evaluationSpotlightEvery month, the subjects in Prescrire’s Spotlight.100 most recent : 1 | 10 | 20 | 30 | 40 | 50 | 60 | 70 | 80 | 90Spotlight100 most recentArchivesRisdiplam (Evrysdi°) in spinalmuscularatrophy. Continue the evaluationFEATURED REVIEWRisdiplam has been authorised in the European Union for use in patients aged 2 months or older with spinalmuscularatrophy. How do Prescrire's editors rate this drug in this situation?Full review (3 pages) available for download by subscribers.Prescrire's rating JUDGEMENT RESERVED
Health technology assessment of the addition of spinalmuscularatrophy (SMA) to the National Newborn Bloodspot Screening Programme Addition of SMA to NNBSP – November 2023 Health Information and Quality Authority Page 1 of 391 Health technology assessment of the addition of spinalmuscularatrophy (SMA) to the National Newborn Bloodspot Screening Programme Published: 27 November 2023 Screening Programme ................................................ 46 2.2 Spinalmuscularatrophy ....................................................................................... 46 2.3 Screening for SMA ............................................................................................... 55 2.4 International practice in newborn bloodspot screening for SMA
Newborn Blood Spot Screening for SpinalMuscularAtrophy ' Phase 2 Newborn Blood Spot Screening for SpinalMuscularAtrophy Phase 2 Health Evidence Review FINAL REPORT JULY 26, 2023 Submitted to: Alberta Health Evidence Reviews Health Evidence and Policy Unit Alberta Health ATB Place North, 18th Floor 10025 Jasper Avenue Edmonton, AB T5J 1S6 Submitted by: The Health Technology & Policy Unit School of Public Health University of Alberta 4-304 Edmonton Clinic Health Academy 11405 - 87 Avenue NW Edmonton, AB T6G 1C9 2 EXECUTIVE SUMMARY SpinalMuscularAtrophy (SMA) is a progressive neurodegenerative disorder resulting from a biallelic deletion within the survival motor neuron 1 (SMN1) gene. Due to the lack of SMN protein production, patients with SMA experience a loss of motor neurons
Onasemnogene abeparvovec (spinalmuscularatrophy) - Benefit assessment according to §35a Social Code Book V 1 Translation of Sections 2.1 to 2.7 of the dossier assessment Onasemnogen-Abeparvovec (spinale Muskelatrophie) – Nutzenbewertung gemäß § 35a SGB V (Version 1.0; Status: 12 August 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. A21-68 Onasemnogene abeparvovec (spinalmuscularatrophy) – Benefit assessment according to §35a Social Code Book V1 Extract of dossier assessment A21-68 Version 1.0 Onasemnogene abeparvovec (spinalmuscularatrophy) 12 August 2021
Risdiplam (spinalmuscularatrophy) - 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 (spinalmuscularatrophy) – Benefit assessment according to §35a Social Code Book V1 Extract of dossier assessment A21-50 Version 1.0 Risdiplam (spinalmuscularatrophy) 29 July 2021 Institute for Quality and Efficiency in Health Care (IQWiG
Onasemnogene abeparvovec (spinalmuscularatrophy) - Addendum to Commission A21-68 1 Translation of addendum A21-128 Onasemnogen-Abeparvovec (spinale Muskelatrophie) – Addendum zum Auftrag A21-68 (Version 1.0; Status: 15 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 15 October 2021 1.0 Commission: A21-128 Version: Status: IQWiG Reports – Commission No. A21-128 Onasemnogene abeparvovec (spinalmuscularatrophy) – Addendum to Commission A21-681 Addendum A21-128 Version 1.0 Onasemnogene abeparvovec – Addendum to Commission A21-68 15 October 2021 Institute for Quality and Efficiency
Risdiplam (spinalmuscularatrophy) - 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 (spinalmuscularatrophy) – 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
Nusinersen (spinalmuscularatrophy) - Benefit assessment according to §35a Social Code Book V 1 Translation of Sections 2.1 to 2.6 of the dossier assessment Nusinersen (spinale Muskelatrophie) – Nutzenbewertung gemäß § 35a SGB V (Version 1.0; Status: 25 February 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. A20-114 Nusinersen (spinalmuscularatrophy) – Benefit assessment according to §35a Social Code Book V1 Extract of dossier assessment A20-114 Version 1.0 Nusinersen (spinalmuscularatrophy) 25 February 2021 Institute for Quality and Efficiency
Nusinersen (spinalmuscularatrophy) - Addendum to Commission A20-114 1 Translation of addendum A21-43 Nusinersen (spinale Muskelatrophie) – Addendum zum Auftrag A20-114 (Version 1.0; Status: 30 April 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 30 April 2021 1.0 Commission: A21-43 Version: Status: IQWiG Reports – Commission No. A21-43 Nusinersen (spinalmuscularatrophy) – Addendum to Commission A20-1141 Addendum A21-43 Version 1.0 Nusinersen – Addendum to Commission A20-114 30 April 2021 Institute for Quality and Efficiency in Health Care (IQWiG) - i - Publishing details Publisher Institute