Friday, 8 May 2026

DMD. Givinostat

 The UK’s National Institute for Health and Care Excellence (NICE) has recommended Givinostat for NHS use in eligible patients with Duchenne muscular dystrophy — a major milestone for families affected by DMD.  

According to the Medscape UK report by Rob Hicks, the recommendation could benefit around 530 patients and may delay disease progression by several years.  

Key points from the announcement:

  • Givinostat (brand name Duvyzat) is approved for boys and young men aged 6+ who are still ambulant when starting treatment.  
  • It is the first non-steroidal treatment approved for all genetic variants of DMD.  
  • Clinical trials showed slower decline in muscle function compared with placebo.  
  • NICE’s draft guidance was issued on 8 May 2026, with final publication expected in June 2026.  

Patient organizations such as Muscular Dystrophy UK and Duchenne UK called the decision a significant breakthrough after years of campaigning for wider access.  

One important caveat: current approval mainly covers ambulant patients. Evidence for people already non-ambulant at treatment initiation is still limited, though additional data collection is ongoing.  


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