Congenital Abnormality

RapidAI Deepens Partnership with Amazon AWS to Expand Clinical Imaging AI Deployment

2 weeks ago talkbio0Tagged Alagille Syndrome, Amazon Web Services, Clearance, clinical AI, Healthcare AI, imaging AI, Lumina 3D, neurovascular AI, Rapid Edge Cloud, RapidAI, RSNA, United States Food and Drug Administration

RapidAI; Amazon Web Services; AWS; clinical AI; imaging AI; RSNA 2025; FDA clearance; Rapid Edge Cloud; healthcare AI; neurovascular AI; Lumina 3D

Rezatapopt Shows Early Promise in TP53 Y220C-Mutant Tumors in PYNNACLE Phase II Trial

2 weeks ago talkbio0Tagged Familial mitral valve prolapse, ORR, ovarian neoplasm, p53 reactivator, Phase 2 Clinical Trials, PYNNACLE trial, Rezatapopt, Solid Neoplasm, Targeted Therapy, TP53 gene, Y220C mutation

Rezatapopt; TP53 Y220C mutation; PYNNACLE trial; solid tumors; phase II clinical trial; ORR; ovarian cancer; p53 reactivator; targeted therapy; PMV Pharmaceuticals

FDA Delays Decision on Ascendis’ Dwarfism Drug by Three Months

2 weeks ago talkbio0Tagged Achondroplasia, Approved, Ascendis Pharma, CNP gene, Deferred, Dwarfism, navepegritide, PDUFA, post-marketing requirement, TransCon, United States Food and Drug Administration

FDA; Ascendis Pharma; TransCon CNP; navepegritide; achondroplasia; dwarfism; PDUFA; post-marketing requirement; delay; approval

PMV Pharma’s p53 Drug Shows Effect in Ovarian Cancer, but Financial Challenges Loom

3 months ago talkbio0Tagged cash runway, Clinical Trials, Familial mitral valve prolapse, File (record), Frequency of Responses, Funding, ovarian neoplasm, TP53 gene, United States Food and Drug Administration

PMV Pharmaceuticals; rezatapopt; p53; ovarian cancer; clinical trial; response rate; FDA filing; funding; cash runway

J&J’s $130M gene therapy bet flunks rare retinal disease trial

7 months ago talkbio0Tagged bota-vec, Disorder of eye, Failure (biologic function), gene therapy, Infrequent, Investments, Johnson and Johnson, loss treatment, Phase 3, Vision, X-linked retinitis pigmentosa

Johnson & Johnson, gene therapy, bota-vec, X-linked retinitis pigmentosa, Phase 3 failure, vision loss treatment, rare eye disease, $130 million investment

Australia Rejects Eisai’s Alzheimer’s Drug Leqembi

9 months ago9 months ago talkbio0Tagged Alzheimer 's disease, Australia, drug rejection, Eisai, Leqembi, Transposition of Great Vessels

Leqembi, Eisai, Alzheimer’s disease, Australia, TGA, drug rejection

Regeneron gene therapy improves hearing in 10 children with rare form of hearing loss

10 months ago talkbio0Tagged Congenital deafness, DB-OTO, gene therapy, Injection of therapeutic agent, otoferlin, Regeneron Pharmaceuticals

Regeneron Pharmaceuticals, DB-OTO gene therapy, otoferlin (OTOF) gene, congenital hearing loss, intracochlear injection

Emerging Competitors in Achondroplasia Treatment: BioMarin’s Dominance Faces New Challenges

11 months ago talkbio0Tagged Achondroplasia, BioMarin, BridgeBio, CNP gene, Disorder of skeletal system, Dwarfism, genetic bone disorders, infigratinib, RBM-007, TransCon, Voxzogo

Achondroplasia, BioMarin, Voxzogo, BridgeBio, infigratinib, TransCon CNP, RBM-007, skeletal disorders, dwarfism, genetic bone disorders.

Healthcare Sector Winners and Losers in the 2024 Government Funding Bill

12 months ago talkbio0Tagged Government, Health care facility, hospital-at-home programs, Legislation, Medicaid, Medicare, pay, Payments, Physicians, shutdown, Symmetrical dyschromatosis of extremities, Telemedicine

Healthcare funding, government shutdown, telehealth, Medicare payments, Medicaid DSH, hospital-at-home programs, physician pay cuts, healthcare legislation.

Philips and AWS Strengthen Strategic AI Partnership, Expanding Cloud-Enabled Diagnostics to Europe

1 year ago talkbio0Tagged Alagille Syndrome, Diagnostics, European (ethnic group), expansion, Generative AI, healthcare innovation, Integrated Diagnostics Portfolio, Philips, Strategic AI Collaboration

Philips, AWS, Strategic AI Collaboration, Cloud-Enabled Diagnostics, European Expansion, Healthcare Innovation, Integrated Diagnostics Portfolio, Generative AI Applications