Regenxbio gives glimpse at early Duchenne gene therapy results, plans pivotal study for accelerated approval

Lei Lei Wu

News Reporter

Regenxbio shared early biomarker results from a 12-year-old boy with Duchenne muscular dystrophy who received a higher dose of its gene therapy, and the biotech …

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Regenxbio gives glimpse at early Duchenne gene therapy results, plans pivotal study for accelerated approval

Lei Lei Wu

News Reporter

Sign up to read this article for free.

Trump names Johns Hopkins researcher Marty Makary to lead the FDA; also names CDC, Surgeon General picks

FDA approves BridgeBio's heart disease drug, setting up competition with Pfizer, Alnylam

Drug or biologic? An Eli Lilly lawsuit could impact weight loss drug compounders

Lilly's board undergoes more changes; Merck strategy chief joins Roger Perlmutter at Eikon

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Re­genxbio gives glimpse at ear­ly Duchenne gene ther­a­py re­sults, plans piv­otal study for ac­cel­er­at­ed ap­proval

Lei Lei Wu

News Reporter

Sign up to read this article for free.

Trump names Johns Hop­kins re­searcher Mar­ty Makary to lead the FDA; al­so names CDC, Sur­geon Gen­er­al picks

FDA ap­proves Bridge­Bio's heart dis­ease drug, set­ting up com­pe­ti­tion with Pfiz­er, Al­ny­lam

Drug or bi­o­log­ic? An Eli Lil­ly law­suit could im­pact weight loss drug com­pounders

Lil­ly's board un­der­goes more changes; Mer­ck strat­e­gy chief joins Roger Perl­mut­ter at Eikon

Sur­round­ed by AI bio gi­ants, tiny start­up Nabla Bio makes de no­vo an­ti­bod­ies look more like drugs

Are pro­grams with com­pound­ed GLP-1s safe for your pop­u­la­tion?

Regenxbio gives glimpse at early Duchenne gene therapy results, plans pivotal study for accelerated approval

Trump names Johns Hopkins researcher Marty Makary to lead the FDA; also names CDC, Surgeon General picks

FDA approves BridgeBio's heart disease drug, setting up competition with Pfizer, Alnylam

Drug or biologic? An Eli Lilly lawsuit could impact weight loss drug compounders

Lilly's board undergoes more changes; Merck strategy chief joins Roger Perlmutter at Eikon

Surrounded by AI bio giants, tiny startup Nabla Bio makes de novo antibodies look more like drugs

Are programs with compounded GLP-1s safe for your population?