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Definitive proof may be lacking that the newly approved Duchenne muscular dystrophy actually works, but most neurologists expect to recommend the medicine for their patients, according to a new survey.

Specifically, 70 percent of the physicians appear fairly convinced that the drug is sufficiently effective to justify its use, and 82 percent expect their patients to start experiencing a benefit within six months, according to the survey of 101 neurologists who treat Duchenne.


The results “confirm the expectation that, given the lack of available options and the devastating nature of the disease, this agent will be widely used and quickly adopted,” wrote RBC analyst Simos Simeonidis in an investor note Tuesday in which he summarized the survey findings conducted by his firm.

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  • my son is DMD and now he is 11 years and he is unable to walk since at the age of 9 years, now he is in wheel chair, his test reports 10 and 11 exons (duplication). Please give suggestion/treatment to my son and save his life.
    thanking you

  • While – “Simeonidis wrote that, since “there are only about 2,000 US boys with DMD (for whom the drug was designed), we do not think this will be the disease US insurance companies will choose to fight the next pricing battle on.” – I think he has missed the point – there will be ‘off-label use’ and that will join the battle. In his own words, Simeonidis said earlier in the post – “given the lack of available options and the devastating nature of the disease, this agent will be widely used and quickly adopted,”. Note those key words – followede by ‘who can put a price on hope’ – I know I am repeating myself in this forum but it bears repeating.

    • Neurologists had best learn the compassionate use procedures. When the required post marketing study fails to prove a survival benefit, as most think will be the outcome, the drug will be withdrawn from market, neurologists will have to get a compassionate use exemption, and the insurance companies will back out. That is the LESSER of the two evils. The other option is not to prescribe the drug with the certainty that you will be sued for malpractice. In that case you will probably lose your career because neurologists as a group carry less than average malpractice insurance because they don’t get sued very often. Appetizing set of choices.

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