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Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a rare, autoimmune neurological disorder in which peripheral nerve demyelination typically results in weakness, impaired limb sensation, fatigue and pain.1–4 CIDP may adversely affect activities of daily living, with a substantial impact on functional ability and psychological well-being.2–6 Primary treatment goals are reducing symptoms, improving functional status and maintaining long-term remission.7 The […]

Michela Guglieri, AAN 2022: Safety and efficacy of corticosteroid regimens for Duchenne muscular dystrophy – Results of an international randomized controlled trial

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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 a randomized, double-blind, parallel-group clinical trial investigating comparative safety and efficacy of different corticosteroid regimens for Duchenne muscular dystrophy.

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 were the aims, design and findings of the study you presented at AAN? (0:17)
  2. What are the implications of these findings for clinical practice? (2:25)

Disclosures: Michela Guglieri has received grant/research support from EC, NIH, PTC, 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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