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316 results

  1. ... for three diseases in animal or cell models: Duchenne muscular dystrophy (in mice); sickle cell disease (in blood stem ...
  2. NLM Digital Collections - Annual report - National Eye Institute (1993) 
    Publication: [Bethesda, Md.] : National Eye Institute
    ... characterization of mutations include Usher syndrome, myotonic dystrophy, Duchenne muscular dystrophy. Long QT syndrome, cataracts, and a variety of ... of the male and female mutation rates in Duchenne muscular dystrophy (DMD). Hum Genet 89:204- 206, 1992. Nickerson ...
  3. NLM Digital Collections - FDA oversight 
    Publication: [Toronto, Ontario] : Thomson Reuters, 20-December-2021
    ... and Co. FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation On February 25, 2021, the FDA granted ... Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation ...
  4. ... A register based system for gene tracking in Duchenne muscular dystrophy. Read AP, et al. J Med Genet 1986 ... A register based system for gene tracking in Duchenne muscular dystrophy. Read AP, et al. J Med Genet 1986 ...
  5. ... SYNDROME DRUG ABUSE DRUG INDUSTRY DRUG SCREENING DRUGS DUCHENNE MUSCULAR DYSTROPHY DUE PROCESS DURATION OF COMMITMENT EASTERN ORTHODOX ETHICS ...
  6. ... should be referred. The important differential diagnosis is Duchenne muscular dystrophy (DMD). A creatine kinase is a good screening test for Duchenne. The necessity to make this diagnosis early is ...
  7. ... Phenylketonuria (PKU) Sickle cell anemia X-linked recessive: Duchenne muscular dystrophy Hemophilia A Autosomal dominant: Familial hypercholesterolemia Marfan syndrome ...
  8. ... protect the male. Diseases such as hemophilia and Duchenne muscular dystrophy occur from a recessive gene on the X ...
  9. ... problem affecting the median nerve in the arm) Duchenne muscular dystrophy (inherited disease that involves muscle weakness) Facioscapulohumeral muscular ...
  10. ... rare diseases: amyotrophic lateral sclerosis, spinal muscular atrophy, Duchenne muscular dystrophy, and Becker muscular dystrophy. 49 In this registry, ...
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