Disease or Syndrome – Page 2

FDA Lifts Hold on Rocket Pharmaceuticals’ Pivotal Gene Therapy Study at Lower Dose

7 days ago talkbio0Tagged C3 complement inhibitor, Capillary Leak Syndrome, Dosage, gene therapy, Glycogen Storage Disease Type IIb, Individual Adjustment, Phase 2 Clinical Trials, Rocket, RP-A501, Study on Hold, United States Food and Drug Administration

FDA; Rocket Pharmaceuticals; gene therapy; Danon disease; clinical hold; RP-A501; dose adjustment; C3 complement inhibitor; capillary leak syndrome; patient death; Phase II trial

American Academy of Pediatrics Breaks with RFK Jr., CDC on COVID Vaccines for Kids

7 days ago talkbio0Tagged Academy of Pediatrics, antiarrhythmic peptide, Centers for Disease Control and Prevention (U.S.), Child, COVID19 (disease), Immunization, Pediatric brand name, Policy, public health guidance, Robert F. Kennedy, Vaccines

American Academy of Pediatrics; AAP; COVID-19 vaccine; children; CDC; Robert F. Kennedy Jr.; pediatric immunizations; public health guidance; vaccine policy

Draft MAHA Commission Report Outlines Broad Vaccine Framework and Addresses Childhood Chronic Disease

7 days ago talkbio0Tagged CDC, Centers for Disease Control and Prevention (U.S.), childhood vaccine schedule, Chronic disease, Commission, draft report, Drug Industry, MAHA, medical freedom, overmedicalization, vaccine framework, vaccine injuries

MAHA Commission; draft report; vaccine framework; childhood vaccine schedule; vaccine injuries; medical freedom; conflicts of interest; CDC; chronic disease; overmedicalization; pharmaceutical industry

Viking Therapeutics Shares Crash After Obesity Pill VK2735 Delivers 12% Weight Loss, But Side Effects and Discontinuations Raise Concerns

7 days ago talkbio0Tagged Adverse event, aspects of adverse effects, Clinical Trials, Contraceptives, Oral, drug efficacy, gastrointestinal, Obesity, Stock Crash, Viking, VK2735, Weight Loss

Viking Therapeutics; VK2735; obesity pill; weight loss; clinical trial; stock crash; side effects; adverse events; gastrointestinal; drug efficacy

FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful

1 week ago talkbio0Tagged Clinical Trials, concerns, Effectiveness, factor IX, Failure (biologic function), Friedreich 's ataxia, Rare Diseases, Rejection, Response process, United States Food and Drug Administration, vatiquinone

PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure

FDA Extends PDUFA Date for Regenxbio’s Hunter Syndrome Gene Therapy RGX-121

1 week ago talkbio0Tagged clemidsogene lanparvovec, Deferred, gene therapy, IDS gene, Infrequent, Mucopolysaccharidosis II, PDUFA, Regenxbio, RGX-121, United States Food and Drug Administration

FDA delay; PDUFA extension; Regenxbio; Hunter syndrome; clemidsogene lanparvovec; RGX-121; gene therapy; Mucopolysaccharidosis II; IDS gene; rare disease

VantAI and Halda Strike $1B+ AI-Driven Partnership on ‘Hold-and-Kill’ Drugs

1 week ago talkbio0Tagged Aortic Valve Insufficiency, cellular targeting, Chimera organism, Drug Discovery, Halda Therapeutics, hold-and-kill, Immunology, Induce (action), Neoplasms, Proximity, RIPTAC, target-effector pairs, VantAI

VantAI; Halda Therapeutics; RIPTAC drugs; hold-and-kill; AI drug discovery; target-effector pairs; oncology; immunology; regulated induced proximity targeting chimeras; $1 billion deal

Battle Intensifies in the Obesity Drug Market as Novo Nordisk and Eli Lilly Vie for Dominance

1 week ago talkbio0Tagged Benefit, Clinical Trials, competition, Compounders, Direct-to-Consumer, eli lilly, Glucagon-Like Peptide 1, manager, Market, Nordisk, Obesity, Pharmaceutical Services, Wegovy, Zepbound

obesity drugs; GLP-1; Wegovy; Zepbound; Novo Nordisk; Eli Lilly; market competition; direct-to-consumer; pharmacy benefit managers; compounders; clinical trials

Stealth BioTherapeutics Resubmits Third FDA Application for Rare Disease Drug

1 week ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, access program, Drug Approval, Elamipretide, Infrequent, resubmission, Stealth BioTherapeutics, United States Food and Drug Administration

Stealth BioTherapeutics; FDA resubmission; rare disease; Barth syndrome; elamipretide; drug approval; expanded access program

Merck KGaA and Skyhawk Therapeutics Launch $2B RNA-Targeting Neurology Collaboration

1 week ago talkbio0Tagged Billion Collaboration, Drug Development, Merck KGaA, milestone payments, nervous system disorder, RNA-targeting, royalties, Skyhawk, SkySTAR platform

Merck KGaA; Skyhawk Therapeutics; RNA-targeting; SkySTAR platform; neurological disorders; $2 billion collaboration; drug development; milestone payments; royalties