Ahead of FDA, EMA recommends authorizing new gene therapy treatment for ultra-rare disease

Paul Schloesser

Associate Editor

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine …

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Ahead of FDA, EMA recommends authorizing new gene therapy treatment for ultra-rare disease

Paul Schloesser

Associate Editor

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Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Paul Schloesser

Associate Editor

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

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

Pa­tient dies in Neu­ro­gene's Rett syn­drome study

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

Ahead of FDA, EMA recommends authorizing new gene therapy treatment for ultra-rare disease

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

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

Patient dies in Neurogene's Rett syndrome study

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