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Washington Post @washingtonpost.com 路 Washington (state) 馃嚭馃嚫路 2w

#GeneEditing #RareDiseases #MedicalInnovation

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David Liu unlocks the power of gene editing to treat rare genetic diseases
A chubby-cheeked baby called KJ made medical history last year. Faced with a life-threatening metabolic disease, KJ's doctors at Children's Hospital of Philadelphia sprinted to create a personalized therapy aimed at fixing the genetic typo at the root of his illness.
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U.S. Food and Drug Administration @fda.gov 路 Silver Spring, Maryland 馃嚭馃嚫路 Jan 5

#PediatricHealth #RareDiseases #DrugApproval

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FDA approves therapy for rare blood disorder in pediatric patients 12
This action marks first drug approved for pediatric patients with acquired thrombotic thrombocytopenic purpura The U. S.
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PEOPLE.com @people.com 路 New York 馃嚭馃嚫路 Jan 4

#CongenitalMyastheniaSyndrome #RareDiseases #HealthAwareness

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'Grey's Anatomy' Actor Steven W. Bailey Reveals Rare Neuromuscular Disorder Diagnosis
CMS is a group of rare hereditary conditions caused by a gene difference that results in muscle weakness, worsening a person's physical ability Grey's Anatomy actor Steven W.