Disease or Syndrome
FDA Halts Kezar’s Lupus Nephritis Trial Due to Safety Concerns Following Four Patient Deaths
FDA, Kezar Life Sciences, lupus nephritis, zetomipzomib, clinical hold, patient deaths, safety concerns
Zealand’s Hypoglycemia Drug Faces Second FDA Rejection Due to Manufacturing Issues
Zealand Pharma, FDA rejection, dasiglucagon, congenital hyperinsulinism, hypoglycemia, CDMO inspection
Biogen’s Higher Dose Spinraza Shows Enhanced Efficacy in Treating Spinal Muscular Atrophy
Biogen, Spinraza, Nusinersen, Spinal Muscular Atrophy (SMA), Higher Dose Regimen, Clinical Trial Results, DEVOTE Study, Motor Function Improvement, Neurodegeneration Reduction
FDA Raises Concerns Over Stealth BioTherapeutics’ Ultra-Rare Disease Drug Approval
Stealth BioTherapeutics, ultra-rare disease, FDA approval, Barth syndrome, elamipretide
Astellas Launches First Direct-to-Consumer Campaign for IZERVAY, Highlighting Its Potential to Slow Geographic Atrophy Progression
IZERVAY, geographic atrophy, age-related macular degeneration, direct-to-consumer campaign, Astellas Pharma
AstraZeneca’s Airsupra Asthma Trial Halted Early Due to Overwhelming Efficacy, Paving Way for Regulatory Submission
AstraZeneca, Airsupra, asthma treatment, clinical trial, overwhelming efficacy, regulatory submission
GEMMABio Secures $100M Partnership to Advance Rare Disease Gene Therapies in Brazil
GEMMABio, gene therapy, Brazil, rare diseases, $100M partnership, health ministry
Merck & Co. Partners with Mestag Therapeutics to Identify Novel Targets for Inflammatory Diseases
Mestag Therapeutics, Merck & Co., Inflammatory diseases, Fibroblast-immune interactions, RAFT platform, Therapeutic targets, Collaboration agreement
Sage Therapeutics’ Dalzanemdor Fails in Alzheimer’s, Hopes Now Rest on Huntington’s Disease Trial
Sage Therapeutics, Dalzanemdor, Alzheimer’s Disease, Huntington’s Disease, Clinical Trials, Neurodegenerative Disorders
Astellas Expands Gene Therapy Portfolio with $2.2 Billion Deal for AviadoBio’s Frontotemporal Dementia Treatment
Astellas Pharma Inc., AviadoBio Ltd., Gene Therapy, Frontotemporal Dementia, FTD-GRN, AVB-101, Progranulin Mutations, Neurodegenerative Diseases