Disease or Syndrome – Page 31

Rethinking Clinical Trial Data in the Era of Decentralization: Key Trends and Challenges (2025)

3 months ago talkbio0Tagged Artificial Intelligence, Clinical Trials, Data Integration, Data Quality, DCT gene, decentralized, Diversity, Preventive monitoring, regulatory guidance, Remote, risk-based oversight, trials

decentralized clinical trials; DCT; data quality; patient diversity; remote monitoring; AI in trials; data integration; risk-based oversight; clinical trial data; regulatory guidance

FDA Investigates Elevidys Death; Sarepta and Roche Deny Link to Gene Therapy

3 months ago talkbio0Tagged Brazil, ELEVIDYS, gene therapy, Market, Muscular Dystrophy, Duchenne, Patient Death, Roche (company), Safety, Suspensions, United States Food and Drug Administration

FDA; Elevidys; Sarepta Therapeutics; Roche; gene therapy; Duchenne muscular dystrophy; patient death; Brazil; market suspension; safety concerns

CHMP Recommends Against Approval of Elevidys Gene Therapy in Europe, Dealing Blow to Sarepta and Roche

4 months ago talkbio0Tagged CHMP, ELEVIDYS, EMBARK trial, Europe, gene therapy, Multiple Acyl Coenzyme A Dehydrogenase Deficiency, Muscular Dystrophy, Duchenne, negative opinion, Roche (company)

CHMP; Elevidys; Sarepta; Roche; gene therapy; Duchenne muscular dystrophy; EMA; negative opinion; Europe; EMBARK trial

Novartis Bets on Covalent Chemistry in $1B Matchpoint Therapeutics Deal

4 months ago talkbio0Tagged Advanced phase, biotechnology partnerships, covalent chemistry, Covalent Interaction, Drug Development, Inflammatory disorder, License, Matchpoint Therapeutics, Novartis, oral inhibitors

Novartis; Matchpoint Therapeutics; covalent chemistry; exclusive option and license agreement; inflammatory diseases; oral inhibitors; Advanced Covalent Exploration platform; drug development; biotechnology partnerships

Rocket Pharmaceuticals Cuts 30% of Staff, Refocuses on Heart Gene Therapies

4 months ago talkbio0Tagged 30%, Anemia, BAG3-associated, cardiovascular gene therapy, Deficiency of pyruvate kinase, Dependovirus, Financial cost, Glycogen Storage Disease Type IIb, Kresladi, layoffs, PKP2-associated cardiomyopathy, restructuring, Rocket

Rocket Pharmaceuticals; layoffs; restructuring; 30% workforce reduction; cardiovascular gene therapy; adeno-associated virus (AAV); Danon disease; PKP2-associated cardiomyopathy; BAG3-associated dilated cardiomyopathy; operating cost reduction; Fanconi Anemia; Pyruvate Kinase Deficiency; KRESLADI

FDA Official Says Sarepta Faces Major Hurdles to Reintroduce DMD Therapy Elevidys

4 months ago talkbio0Tagged approval status, CBER, concerns, ELEVIDYS, gene therapy, Muscular Dystrophy, Duchenne, Pauses, Safety, Scientific Study, shipment, United States Food and Drug Administration

Sarepta; Elevidys; FDA; Duchenne muscular dystrophy; gene therapy; CBER; safety concerns; shipment pause; new studies; approval status

AstraZeneca’s Self-Administered Myasthenia Gravis Drug Gefurulimab Clears Phase 3 Trial

4 months ago talkbio0Tagged Acquisition, Alexion, AstraZeneca, Autoimmune Diseases, C5 inhibitor, Clinical Trials, Gefurulimab, Myasthenia Gravis, PREVAIL trial, self-administered drug

AstraZeneca; gefurulimab; myasthenia gravis; self-administered drug; Phase 3 trial; PREVAIL trial; C5 inhibitor; autoimmune disease; clinical trial; Alexion acquisition