26.07.23
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”...
Read more30.06.23
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...
Read more29.06.23
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).
Read more13.06.23
19.05.23