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NEW YORK and CLEVELAND – Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced the activation of a second clinical trial site in its pivotal Phase 3 VIITAL™ study of its investigational EB-101 treatment for recessive dystrophic epidermolysis bullosa (RDEB) at UMass Memorial Medical Center... -
AMSTERDAM, Netherlands and LOUISVILLE, Ky. – NORGINE B.V. (Norgine) a leading European specialist pharmaceutical company and US WorldMeds (USWM), a Kentucky-based specialty pharmaceutical company, today announced an exclusive licensing agreement by which Norgine will register and commercialise difluoromethylornithine, DFMO (eflornithine) in Europe, Commonwealth of Independent States, Australia and New Zealand. DFMO is a repurposed molecule investigated for use...
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GRAND RAPIDS, Mich. – Tetra Therapeutics, a clinical stage biopharmaceutical company focused on the development of compounds for the treatment of brain disorders associated with cognitive and memory deficits, notably BPN14770, a selective inhibitor of phosphodiesterase‐4D (PDE4D), announced changes to its executive team as the company marks one year since...
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Rare disease spotlight
Rare disease drugmakers to Congress: Don't gut the accelerated approval pathway http://dlvr.it/S4yXD5
Ballet star, 18, who has cystic fibrosis lands place at leading dance school https://www.dailymail.co.uk/news/article-9854281/Ballet-star-18-cystic-fibrosis-lands-place-leading-dance-school.html