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Huntington’s disease (HD) is a neurodegenerative disease inherited in an autosomal dominant manner. It is caused by an expansion of cytosine, adenine, guanine (CAG) repeats within the huntingtin (HTT) gene, which is located on chromosome 4. This pathological expansion of CAG repeats results in the production of a mutant huntingtin protein with an abnormally long polyglutamine […]

Michela Guglieri, AAN 2022: Unmet needs and the role of corticosteroids in the treatment paradigm of Duchenne muscular dystrophy

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Published Online: Apr 19th 2022

Corticosteroids can improve muscle function in boys with Duchenne muscular dystrophy and are commonly prescribed for newly diagnosed patients. Dr Michela Guglieri (Newcastle University, Newcastle upon Tyne, United Kingdom) discusses the current unmet needs in DMD, and the role of corticosteroids in the treatment paradigm of DMD.

The abstract entitled ‘Comparative Safety and Efficacy of Different Corticosteroid Regimens for Duchenne Muscular Dystrophy: Results of an International Randomized Controlled Trial’ was presented at the American Academy of Neurology (AAN) annual meeting, April 2-7, 2022.

Questions: 

  1. What are the current unmet needs in the treatment of Duchenne muscular dystrophy (DMD)? (0:15)
  2. What is the role of corticosteroids in the treatment paradigm for DMD? (1:45)

Disclosures: Michela Guglieri has received grant/research support from EC, NIH, PTC, and Sarepta; and has served on the advisory board for Dyne, Pfizer and NS Pharma; and has received honoraria/honorarium from Sarepta.

Support: Interview and filming supported by Touch Medical Media. Interview conducted by Katey Gabrysch.

Filmed as a highlight of AAN 2022

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