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Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder of upper and lower motor neurons that results in progressive motor impairment. ALS is the most common disease of motor neurons with an annual incidence of approximately 1.7–2.5 per 100,000 people. It is a terminal condition with a typical life expectancy of 2–5 years from symptom onset. […]

John Bodensteiner, AAN 2018 – Expert perspective on Duchenne muscular dystrophy

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Published Online: May 2nd 2018

John Bodensteiner (Consultant, Child and Adolescent Neurology), our Paediatric Neurology Section Editor for US Neurology, shares his expert perspective on Duchenne muscular dystrophy.

Questions

1. What do you consider the most promising current and emerging treatment options for Duchenne Muscular Dystrophy (DMD)? (0:11)
2. Could you tell us a little about the CINRG Duchenne Natural History Study? (0:34)
3. What were the findings of the CINRG study on the long-term effects of glucocorticoids in patients with DMD? (1:52)
4. On the basis of current evidence, what do you consider to be the optimal treatment regimen for children with DMD? (3:09)

Speaker disclosure: John Bodensteiner has nothing to disclose in relation to this video interview.

 

Filmed at the American Academy of Neurology (AAN) Annual Meeting 2018, Los Angeles, CA, US, April 2018.

 

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