Status In progress
Technology type Medicine
Decision Selected
Reason for decision Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
Process HST
ID number 4051

Provisional Schedule

Committee meeting 09 February 2023
Expected publication 25 May 2023

Project Team

Project lead Celia Mayers

Email enquiries


Companies sponsors Novartis Gene Therapies (onasemnogene abeparvovec)
Others Department of Health and Social Care
  European Alliance for Newborn Screening in Spinal Muscular Atrophy
  NHS England
Patient carer groups Ally Cadence Trust for Spinal Muscular Atrophy
  Arthritis and Musculoskeletal Alliance
  Brain and Spine Foundation
  Brain Charity
  Disability Rights UK
  Genetic Alliance UK
  Leonard Cheshire Disability
  Muscular Dystrophy UK
  National Children’s Bureau
  Neurological Alliance
  South Asian Health Foundation
  Specialised Healthcare Alliance
  Spinal Muscular Atrophy UK (SMA UK)
  The Annabelle Rose Foundation for Spinal Muscular Atrophy
  Together for Short Lives
Professional groups Association of Anaesthetists
  Association of British Neurologists
  Association of Genetic Nurses and Counsellors
  Association of Paediatric Anaesthetists of Great Britain and Ireland
  Association of Surgeons of Great Britain and Ireland
  British Dietetic Association
  British Institute of Musculoskeletal Medicine
  British Myology Society
  British Neuropathological Society
  British Orthopaedic Association
  British Paediatric Neurology Association
  British Society for Children's Orthopaedic Surgery
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  British Society for Human Genetics
  British Society of Rehabilitation Medicine
  Chartered Society for Physiotherapy
  Institute of Neurology
  Neonatal & Paediatric Pharmacists Group
  Primary Care and Community Neurology Society
  Royal College of Anaesthetists
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics & Child Health
  Royal College of Pathologists
  Royal College of Physicians
  Royal College of Surgeons
  Royal Pharmaceutical Society
  Royal Society of Medicine
  SMA Reach
  UK Clinical Pharmacy Association
  UK Genetic Testing Network
Associated public health groups Public Health Wales
  UK Health Security Agency
Comparator companies None
General commentators All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  Board of Community Health Councils in Wales
  British National Formulary
  Care Quality Commission
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  National Services Division
  Neurological Alliance of Scotland
  NHS Alliance
  NHS Confederation
  Scottish Medicines Consortium
  Wales Neurological Alliance
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups Cochrane Cystic Fibrosis & Genetic Disorders Group
  Cochrane Neuromuscular
  Genomics England
  John Walton Muscular Dystrophy Research Centre
  MRC Centre for Neuromuscular Diseases
  MRC Clinical Trials Unit
  National Institute for Health Research


Key events during the development of the guidance:

Date Update
09 June 2022 Invitation to participate
22 April 2022 - 23 May 2022 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators
05 May 2022 In progress

For further information on our processes and methods, please see our CHTE processes and methods manual