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The Wall Street Journal News source @wsj.com 路 New York City 馃嚭馃嚫路 2d

#FDA #AcceleratedApproval #RareDiseases

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Opinion | Who's in Charge at the FDA?
Commissioner Marty Makary talks about accelerated cures while biologics chief Vinay Prasad quietly scuttles them. Food and Drug Administration chief Marty Makary boasted on these pages Monday about a new effort to allow more flexible reviews for drugs treating rare diseases.
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New York Post News source @nypost.com 路 New York City 馃嚭馃嚫路 3d

#RareDiseases #Biotechnology #HealthcareReform

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How Trump's FDA is breaking his promise to America's patients
Months into his second term, President Donald Trump's top health officials were clear. They promised to focus on "cures" and "meaningful treatments.
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NPR News source @npr.org 路 Washington (state) 馃嚭馃嚫路 3d

#RareDiseases #GeneEditing #PersonalizedMedicine

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The FDA creates a quicker path for gene therapies
The Food and Drug Administration Monday unveiled the details of a new policy designed to make it easier and quicker for patients with very rare diseases to get cutting-edge treatments.
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Washington Post News source @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 News source @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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U.S. Food and Drug Administration News source @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.
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PEOPLE.com News source @people.com 路 New York 馃嚭馃嚫路 Jan 4

#CongenitalMyastheniaSyndrome #RareDiseases #HealthAwareness

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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.