Coronary artery bypass surgery provides long-term benefits for children whose hearts and blood vessels are damaged by Kawasaki disease, Japanese researchers report in Circulation: Journal of the American Heart Association. Researchers followed 114 people for up to 25 years who had bypass surgery as children or adolescents (ages 1 to...
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Tucson, Arizona — Critical Path Institute’s (C-Path) Duchenne Regulatory Science Consortium (D-RSC) is excited to announce the launch of a groundbreaking model-based Clinical Trial Simulator (CTS), specifically designed to improve design of efficacy studies for potential therapies for Duchenne muscular dystrophy (DMD). This pioneering Drug Development Tool is set to...
TUCSON, Ariz. — Critical Path Institute (C-Path), a leader in accelerating drug development for rare diseases, today announced the targeted integration of additional Friedreich’s ataxia (FA) datasets into C-Path’s Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) as part of a partnership with Friedreich’s Ataxia Research Alliance (FARA). This update...
Tucson, Arizona — Critical Path Institute (C-Path) today announced the formation of a new task force under its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®), dedicated to advancing therapeutic development for Progressive Supranuclear Palsy (PSP). This initiative brings together leading organizations and experts in a concerted effort to tackle...
Tucson, AZ. – Critical Path Institute (C-Path) is pleased to announce the release of a new peer-reviewed publication, titled “Transforming Drug Development for Neurological Disorders: Proceedings from a Multi-disease Area Workshop,” now published in Neurotherapeutics, The Journal of the American Society for Experimental Neurotherapeutics. A distinguished team of C-Path scientists...
PHILADELPHIA, PA — Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human...
First cleared CD19-CAR T IND application for generalized myasthenia gravis announced in the U.S. and fourth IND clearance for CABA-201 across a broad range of autoimmune diseases Clearance expands the clinical development of CABA-201 beyond rheumatology into neurology Phase 1/2 clinical trial evaluating CABA-201 in generalized myasthenia gravis features...
Research Triangle Park, N.C. — BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) announced that the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has issued a positive recommendation for ORLADEYO® (berotralstat) to be reimbursed for the routine prevention of attacks of hereditary angioedema (HAE) in adults...
BASKING RIDGE, N.J. – Caladrius Biosciences, Inc. (“Caladrius” or the “Company”), a clinical-stage biopharmaceutical company dedicated to the development of cellular therapies designed to reverse disease, today announced that the U.S. Food and Drug Administration (“FDA”) has granted orphan drug designation to the Company’s CD34+ cell therapy product, CLBS12, for...
Rochester, NY – New research has identified the specific biological mechanism behind the muscle dysfunction found in myotonic dystrophy type 1 (DM1) and further shows that calcium channel blockers can reverse these symptoms in animal models of the disease. The researchers believe this class of drugs, widely used to treat...