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(Reuters) -A young patient died due to cardiac arrest after receiving Pfizer's experimental gene therapy being tested in a mid-stage trial for a muscle-wasting disorder called Duchenne muscular ...
Duchenne muscular dystrophy is the most common type of muscular dystrophy, [3] with a median life expectancy of 27–31 years. [5] [11] However, with comprehensive care, some individuals may live into their 30s or 40s. [3] Duchenne muscular dystrophy is considerably rarer in females, occurring in approximately one in 50,000,000 live female ...
Melanie Sanford's son Hudson was diagnosed with Duchenne, a fatal, progressive form of muscular dystrophy. She was told Hudson would only live until age 28 until she found hope in a breakthrough ...
X-linked recessive conditions include the serious diseases hemophilia A, Duchenne muscular dystrophy, and Lesch–Nyhan syndrome, as well as common and less serious conditions such as male pattern baldness and red–green color blindness.
Darius Goes West: The Roll of his Life is a documentary film by Logan Smalley about Darius Weems, a teenager living with Duchenne muscular dystrophy.In the middle of 2005 Weems embarked on a 7,000 mile road trip across the United States from his hometown in Georgia to MTV Headquarters in Los Angeles to ask them to customize his wheelchair on Pimp My Ride, as well to promote awareness of the ...
(Reuters) -Pfizer said on Wednesday its experimental gene therapy for Duchenne muscular dystrophy (DMD) failed to improve the motion function of patients in a late-stage trial compared with a placebo.
Duchenne muscular dystrophy. P – Point mutation, or any insertion/deletion entirely inside one gene; D – Deletion of a gene or genes; Dup - Duplication of a gene or genes; C – Whole chromosome extra, missing, or both (see chromosome abnormality) T – Trinucleotide repeat disorders: gene is extended in length
Many affected people will eventually become unable to walk [2] and Duchenne muscular dystrophy in particular is associated with shortened life expectancy. Muscular dystrophy was first described in the 1830s by Charles Bell. [2] The word "dystrophy" comes from the Greek dys, meaning "no, un-" and troph-meaning "nourish". [2]
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