News

Advancing Rare Disease Research: Zevra’s Acquisition of Acer Therapeutics

Community Statement from Kisbee Therapeutics [30th August 2023]

Should patients with rare neurodegenerative diseases have the "right to try" experimental treatments? 

Aisling Cantwell, a MSc Drug Development Science student at King’s College London, would like to invite you to complete the following questionnaire, which aims to increase understanding of the views of rare disease caregivers on the principle of the “right to try”...

Cyclo Therapeutics Announces Publication of Positive Data from Phase 2 Clinical Study of Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C1

Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with diseases, today announced the publication of positive data from its Phase 1/2 clinical trial, which demonstrated promising safety and efficacy results for Trappsol® Cyclo™ in the treatment of Niemann-Pick Disease Type C1, a rare, genetic disease causing cholesterol accumulation in cells, leading to dysfunction of the liver, lung, spleen and brain and premature death...

IntraBio Announces Positive Pivotal Trial Results of IB1001 for the Treatment of Niemann-Pick Disease Type C

IntraBio Inc today announced positive topline results from the pivotal, Phase III IB1001-301 clinical trial (NCT05163288), showing IB1001 significantly improved symptoms, functioning, quality of life, and cognition in pediatric and adult patients with Niemann-Pick disease Type C (NPC).