W
Washington Post @washingtonpost.com 路 Washington (state) 馃嚭馃嚫路 1w

#GeneEditing #RareDiseases #MedicalInnovation

Link Preview
www.washingtonpost.com
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.
Avatar
Yahoo! @ca.style.yahoo.com 路 Sunnyvale, California 馃嚭馃嚫路 3w

#ChildhoodDementia #SanfilippoSyndrome #RareDisease

Link Preview
ca.style.yahoo.com
Next 365 days could be crucial in my five-year-old son's dementia fight
Add Yahoo as a preferred source to see more of our stories on Google. The next year could be crucial in a five-year-old's battle with a rare form of childhood dementia, his mother believes.
F
U.S. Food and Drug Administration @fda.gov 路 Silver Spring, Maryland 馃嚭馃嚫路 Jan 5

#PediatricHealth #RareDiseases #DrugApproval

Link Preview
www.fda.gov
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.
F
U.S. Food and Drug Administration @fda.gov 路 Silver Spring, Maryland 馃嚭馃嚫路 Jan 5

#PediatricHealth #RareDiseases #DrugApproval

Link Preview
www.fda.gov
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.
Avatar
PEOPLE.com @people.com 路 New York 馃嚭馃嚫路 Jan 4

#CongenitalMyastheniaSyndrome #RareDiseases #HealthAwareness

Link Preview
people.com
'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.