Status In progress
Process STA 2018

Project Team

Project lead Jo Ekeledo

Email enquiries


Companies sponsors Santhera Pharmaceuticals (idebenone)
Others Department of Health
  NHS England
  NHS South Cheshire CCG
  NHS West London CCG
  Welsh Government
Patient carer groups Action on Pain
  Action Duchenne
  Action for Sick Children
  Alex's Wish
  Arthritis & Musculoskeletal Alliance
  Contact a Family
  Disability Rights UK
  Duchenne Family Support Group
  Duchenne Now
  Duchenne Research Fund
  Duchenne UK
  Genetic Alliance UK
  Harrison’s Fund
  Information Advice and Support Service Network
  Leonard Cheshire Disability
  Muscular Dystrophy UK
  Muslim Council of Britain
  National Children’s Bureau
  South Asian Health Foundation
  Specialised Healthcare Alliance
  Together for Short Lives
Professional groups Association of Anaesthetists of Great Britain & Ireland
  Association of British Neurologists
  Association of Genetic Nurses & Counsellors
  Association of Respiratory Nurse Specialists
  Association of Surgeons of Great Britain and Ireland
  British Cardiovascular Society
  British Dietetic Association
  British Institute of Musculoskeletal Medicine
  British Orthopaedic Association
  British Paediatric Neurology Association
  British Paediatric Respiratory Society
  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
  British Thoracic Society
  Chartered Society of Physiotherapy
  Primary Care Respiratory Society UK
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics and Child Health
  Royal College of Pathologists
  Royal College of Physicians
  Royal College of Surgeons
  Royal Pharmaceutical Society
  Royal Society of Medicine
  UK Genetic Testing Network
  UK Clinical Pharmacy Association
  The National Congenital Anomaly and Rare Disease Registration Service
  The North Star Project


Associated public health groups Public Health England
  Public Health Wales
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
  NHS Alliance
  NHS Commercial Medicines Unit
  NHS Confederation
  Scottish Medicines Consortium
  National Services division for Scotland
  Welsh Health Specialised Services Committee
Relevant research groups British Myology Society
  Cochrane Cystic Fibrosis and Genetic Disorders Group
  Cochrane Musculoskeletal Group
  John Walton Centre for Muscular Dystrophy (Newcastle University)
  MRC Centre for Neuromuscular Diseases
  MRC Clinical Trials Unit
  National Institute for Health Research


Key events during the development of the guidance:

Date Update
18 March 2020 As you will be aware, the Department for Health and Social Care has asked NICE to carry out a Single Technology Appraisal of Puldysa (idebenone). Please note that following on from advice received from the company this appraisal has been rescheduled. Therefore, we now anticipate that the appraisal will begin after the summer of 2020.
20 November 2019 - 18 December 2019 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
22 November 2019 In progress, In progress
22 February 2018 Suspended, Suspended
10 March 2017 (10:00) Scoping workshop (London)
19 January 2017 - 16 February 2017 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

For further information on how we develop guidance, please see our page about NICE technology appraisal guidance