#RareDiseases #MPS #FDAApproval
www.newsweek.com
Moms stage "funeral" outside FDA in drug protest
More than 100 mothers and advocates affected by a type of rare disease gathered outside the Food and Drug Administration (FDA) on Wednesday to stage a funeral to "mourn the futures" of their children, following the agency's rejections of various rare disease treatments.
#RareDiseases #Biotech #DrugDevelopment
fortune.com
The $3.4 billion lesson Big Pharma needs to learn: its shelved drugs could save millions of patients | Fortune
For patients living with rare and neglected diseases, the next breakthrough treatment might already be sitting in a pharmaceutical company's filing cabinet.
#RareDiseases #FDAApproval #PatientAdvocacy
www.newsweek.com
Mothers "terrified" as children could lose treatments after FDA denials
Mothers from across the country have spoken to Newsweek about their concern for their children after the Food and Drug Administration (FDA) has been rejecting various treatments for rare diseases that can be life-threatening.
#FDA #RareDiseases #HealthPolicy
www.newsweek.com
Republican launches investigation into Trump admin FDA denials
Senator Ron Johnson has launched an investigation into the Food and Drug Administration (FDA) over the agency's recent denials of treatments for rare diseases, the Wisconsin Republican said in an interview with Bloomberg.
W
#FDA #AcceleratedApproval #RareDiseases
www.wsj.com
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.
W
#GeneEditing #RareDiseases #MedicalInnovation
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.
#CongenitalMyastheniaSyndrome #RareDiseases #HealthAwareness
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.
