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Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate
Associated Press via AOL· 12 hours agoRobin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited...
Sarepta Gains as FDA Official Overrules Staff to Approve Therapy
Bloomberg via Yahoo Finance· 59 minutes ago(Bloomberg) -- A top US Food and Drug Administration official overrode reviewers to give broad...
Sarepta Catapults 39% After Hitting A 'Grand Slam' Approval In Muscular Dystrophy
Investor's Business Daily· 2 hours agoSarepta stock rocketed Friday after hitting a "grand slam" approval that expands the market for its...
Sarepta Skyrockets After FDA Signs Off On Duchenne Gene Therapy
Investor's Business Daily· 5 hours agoLast year, the FDA granted the Sarepta Therapeutics (SRPT) gene therapy, dubbed Elevidys, an accelerated approval for children ages 4 and 5 with Duchenne ...
Sarepta Stock Surges on Expanded Elevidys Approval. It’s a ‘Best-Case’ Scenario for Shares.
Barrons.com· 1 hour agoThe biopharmaceutical company says the drug was approved for ambulatory and non-ambulatory people,...
FDA expands approval of first gene therapy for rare form of muscular dystrophy
CNN via AOL· 17 hours agoThe US Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most ...
US FDA approves expanded use of Sarepta’s Duchenne gene therapy
WHTC 1450 Holland· 18 hours ago(Reuters) -The U.S. Food and Drug Administration allowed the expanded use of Sarepta Therapeutics' ...
Sarepta Duchenne gene therapy wins broader use from FDA
BioPharma Dive via Yahoo Finance· 23 hours agoThe approval makes Elevidys available to most Duchenne patients at least 4 years of age, despite...
Investigative Sickle Cell gene therapy patient advocates for change on Capitol Hill
FOX 8 Cleveland· 18 hours ago22-year-old Canaan Evans has been living with Sickle Cell disease his entire life, but thanks to a...
Decades of gene therapy research pay off big for Madison company
Wisconsin State Journal· 23 hours ago“From the very start, we wanted to focus on gene therapy,” said Jim Hagstrom, a molecular biologist...