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Tag: rare disease

Inventiva annonce la nomination de Jason Campagna en tant que Président de la R&D et Directeur Médical et de Martine Zimmermann en tant que...


Daix (France), New York City (New York, États-Unis), le 9 juillet 2025
– Inventiva (Euronext Paris et Nasdaq : IVA) (« Inventiva » ou la « Société »), société biopharmaceutique spécialisée dans le développement de thérapies orales pour le traitement de la stéatohépatite associée à un dysfonctionnement métabolique (« MASH »), a annoncé aujourd'hui une transition au sein de son équipe de direction avec la nomination de Jason Campagna, MD, PhD, en tant que Président de la Recherche et du Développement (« R&D ») et Directeur Médical (« CMO »), ainsi que Martine Zimmermann, PharmD, en tant que Vice-Présidente Exécutive (« EVP ») en charge des affaires réglementaires et de l’ assurance qualité.

Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis

Data from Orbit and Cosmic studies expected around the end of the year
Data from Orbit and Cosmic studies expected around the end of the year

A Life Rewritten: KFSHRC Treats Rare Disease After 22 Years of Specialized Care

RIYADH, Saudi Arabia, July 09, 2025 (GLOBE NEWSWIRE) -- In a rare example of a lifelong bond between a patient and a healthcare institution, King Faisal Specialist Hospital & Research Centre (KFSHRC) in Riyadh has continued to support a patient since infancy, providing 22 years of continuous care for a rare genetic blood clotting disorder, ultimately culminating in a liver transplant—the first of its kind globally for this particular condition.

U.S. FDA Grants Orphan Drug Designation to Adcentrx Therapeutics’ ADRX-0405 STEAP1 ADC for Gastric Cancer

Orphan drug designation highlights the potential for ADRX-0405 to address the high unmet need in gastric cancerSAN DIEGO, July 8, 2025 /PRNewswire/ -- Adcentrx Therapeutics ("Adcentrx"),...

Chan Zuckerberg Initiative, Innovative Genomics Institute Announce New Center for Pediatric CRISPR Cures

SAN FRANCISCO, July 8, 2025 /PRNewswire/ -- Personalized CRISPR cures for children born with rare genetic diseases are now a step closer to being more...

Hansa Biopharma appoints Dr Richard Philipson as Chief Medical Officer

LUND, Sweden, July 8, 2025 /PRNewswire/ --  Hansa Biopharma AB, "Hansa" (NASDAQ Stockholm: HNSA) today announced that Dr. Richard Philipson has been appointed Chief...

Hansa Biopharma appoints Dr Richard Philipson as Chief Medical Officer

LUND, Sweden, July 8, 2025 /PRNewswire/ --  Hansa Biopharma AB, "Hansa" (NASDAQ Stockholm: HNSA) today announced that Dr. Richard Philipson has been appointed Chief...

INOVIO to Present at Upcoming Scientific Conference

PLYMOUTH MEETING, Pa., July 7, 2025 /PRNewswire/ -- INOVIO (NASDAQ:INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and...

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