Research and Treatments Archives - Walking Strong https://walkingstrong.org/category/research-and-treatments/ Together, We Can Cure Duchenne Muscular Dystrophy Thu, 22 May 2025 13:05:56 +0000 en hourly 1 https://walkingstrong.org/wp-content/uploads/2018/04/favicon-150x150.png Research and Treatments Archives - Walking Strong https://walkingstrong.org/category/research-and-treatments/ 32 32 Targeted Therapies for DMD https://walkingstrong.org/targeted-therapies-for-dmd/ https://walkingstrong.org/targeted-therapies-for-dmd/#respond Thu, 01 May 2025 19:36:55 +0000 https://walkingstrong.cmsnc.website/?p=11437 Targeted Therapies Aim at the Root of the Disease At the 2025 Muscular Dystrophy Association Clinical & Scientific Conference in Dallas, Dr. Matthew Alexander, associate professor of pediatric neurology and genetics at the University of Alabama, Birmingham, led a compelling session on the future of muscular dystrophy treatment. The focus? Targeted therapies are designed to […]

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]]> https://walkingstrong.org/targeted-therapies-for-dmd/feed/ 0 FDA Finally Approves First Ever Treatment Drug – Finally! https://walkingstrong.org/fda-finally-approves-first-ever-treatment-drug-finally/ https://walkingstrong.org/fda-finally-approves-first-ever-treatment-drug-finally/#respond Tue, 27 Sep 2016 20:53:40 +0000 https://walkingstrong.org/?p=6547 100 days after the FDA’s legally mandated decision date,the FDA announced accelerated approval for  Eteplirsen by Sarepta Therapeutics.  Eteplirsen is the first therapy drug approved for Duchenne which works to skip over the deleted exons.  The approval did come without bureaucratic “drama” and in-fighting.  There were definite opponents for approval, based on the nonsense of […]

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]]> https://walkingstrong.org/fda-finally-approves-first-ever-treatment-drug-finally/feed/ 0 FDA – NOT FOR DUCHENNE https://walkingstrong.org/fda-not-for-duchenne/ https://walkingstrong.org/fda-not-for-duchenne/#respond Tue, 26 Apr 2016 17:28:45 +0000 https://walkingstrong.org/?p=6424 April 26, 2016   As I sit bleary-eyed  on the airplane flying home from what should have been a “successful” trip to DC, I am still stunned and disheartened from yesterday after a grueling TWELVE HOUR meeting with the FDA and it’s Advisory Committee, that went from an historic event into a catastrophe.   I want […]

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]]> https://walkingstrong.org/fda-not-for-duchenne/feed/ 0 Clinical Trial – Day 1 https://walkingstrong.org/clinical-trial-day-1/ https://walkingstrong.org/clinical-trial-day-1/#respond Thu, 09 Jul 2015 18:21:04 +0000 https://walkingstrong.org/?p=6416 I guess you can say – “we are lucky.’   After 5 days of putting Alexander through extensive testing (a 3 hour MRI, extensive bloodwork, ECG, Pulmonary, 2 days of physical therapy assessment,   fecal testing (ek!) and a physical exam), we received official word last night that he was accepted into a clinical trial for a […]

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]]> https://walkingstrong.org/clinical-trial-day-1/feed/ 0 CLINICAL TRIAL – No easy choices https://walkingstrong.org/clinical-trial-no-easy-choices/ https://walkingstrong.org/clinical-trial-no-easy-choices/#respond Wed, 11 Feb 2015 16:11:07 +0000 https://walkingstrong.org/?p=6288 On a Saturday morning at 9:00am I received a call from our Neurologist at UCLA. He called to tell me the news that there is a clinical trial at UCLA that Alexander could qualify for. After a 45 min conversation, I was left hopeful but at the same time anxious and a little sad. The […]

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