Improving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA) (SMA-REACH)

 Skeletal / Posted 3 weeks ago

The SMA REACH UK Network is a national and international partnership between doctors and therapists involved in the care of children and adults with Spinal Muscular Atrophy. This network is supported by Biogen and SMA UK.

  • Inclusion Criteria : Ages Eligible for Study: up to 99 Years (Child, Adult, Older Adult) Sexes Eligible for Study: All Accepts Healthy Volunteers: No It will be genetically confirmed SMA
  • Exclusion Criteria : Involvement in clinical trials is not an exclusion criterion nor having had surgical procedures. Patients who are participating in clinical trials with novel treatments will also be included in the database although the data from this subgroup won't be analysed in the natural history study, nor shared with pharmaceutical companies and other third parties as part of the ISMAC collaboration.
  • Study end date : 28/02/2022
  • Principal Investigator : Francesco Muntoni
Contact details

Great Ormond Street Hospital for Children NHS Foundation Trust

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