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An experimental gene therapy for Duchenne muscular dystrophy, licensed to Sarepta Therapeutics, produced jaw-dropping increases in a crucial muscle protein normally missing in patients with the disease, according to preliminary clinical trial data released Tuesday.

The data were collected from just three boys, but the effect of the gene therapy — producing 38 percent of a truncated form of the normal dystrophin muscle protein — is profound enough to suggest it may halt or even reverse the effects of Duchenne in certain patients.

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  • I would hope and pray that a similar drug could be developed for treatment of ALS. My father passed away from the effects of this dreaded disease, and it appears that it is becoming more prevalent, with no cure in sight.

  • This is the story that just makes you weep….so happy for the kids and their parents, its a glimmer of hope for them and for all us parents of special needs kids who dream that gene therapy can work for them too.I pray for the moment when my 30 year old son looks into my eyes and says …”.Hi Mom. I love you!!”

    Trumps signing the law that allows families with desperate fatal no option diseases to participate in earlier than usual trials may seriously pay off after all.

  • My daughter is affected with myosin storage myopathy. Is there any drug for this type. Pls help her

  • I’m very interested inrolling.I’m this trial. HE GETs weekly infusions of the exondise

  • Sir My son in laws have the DMD could please guide me when and where can this medication and how to be treated ?

  • My son Shivam Anjay age 20 years is patient of DMD. His 46 to 50 economy found delete and economy 45 & exon 51 is present. If, he may be consider for Sarepta’s Gene Therapy, please guide us to save the life of my son Shivam Anay.

  • thank you for the information, hoping to see the commercial verient in market soon, beleive me you are doing great job!! God bless you

  • Drugs that offer cures to acknowledged fatal diseases should be extracted from the economic system that makes them too expensive to use liberally, which strikes me as perverse as far as research goes anyway, and ‘adopted’ in some form by the State. The profit motive puts a flywheel on progress, and a deterrent as well.

    • If they show serious results insurances will cover it, and theres always a Go Fund Me Page. I agree drugs are expensive, but researchers tend not to want to work for 20 years on something without being paid.

  • Mi hijo Alvaro Exequiel tiene 9 años Argentino , le diagnosticaron DMD , todavía camina y yo veo que la enfermedad avanza rápidamente , porfavor nesecito contactarme con uds , su nivel de CK es de 7000 . Respondanme gracias.

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