Rafael Holdings Announces Presentation of Preliminary Data from Ongoing Phase 3 TransportNPC™ Open-Label, Single-Arm Sub-Study in Patients Less Than 3 Years Old

First data on treatment of NPC1 with Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin) in this age group over a period of 48 weeks At 48 weeks of the sub-study, 7 of 9 patients1 showed stabilization or improvement in CGI-S score Data presented at the15th International Congress of Inborn Errors of Metabolism (ICIEM) NEWARK, N.J., Sept. 16, 2025 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc. (NYSE: RFL; NYSE American: RFL-WT) announced today that data from Cyclo Therapeutics’ Phase 3 TransportNPC™ open-label, sin ...