Updates

Cyclo Therapeutics Provides Update on Trappsol® Cyclo™ Development Programme

ASMD Project – The Invisible Years – Survey

Proposed Patient Organisation Recruitment Communication At Niemann-Pick UK, we know that the road to an ASMD diagnosis is often not straightforward. Many individuals and families experience years of uncertainty, unanswered questions, and repeated visits to healthcare professionals before finally...

Zevra Therapeutics Announces Publication of Four-Year Real-World Data from U.S. Early Access Program Evaluating MIPLYFFA® (arimoclomol) in Niemann-Pick Disease Type C (NPC)

Zevra Therapeutics Announces Publication of Positive MIPLYFFA® Data from Pediatric Substudy in Infants with Niemann-Pick Disease Type C (NPC)

Global Milestone Reached in Niemann-Pick Type C Clinical Research

Advancing Research for the Global NPC Community The International Niemann-Pick Disease Alliance (INPDA) is pleased to share an important update from Cyclo Therapeutics, marking a significant milestone in global research efforts for Niemann-Pick disease type C (NPC). Cyclo Therapeutics...

Beren Therapeutics Secures $300M Financing to Support the Potential Commercial Launch of Adrabetadex and Long-Term Care Initiatives for Infantile-Onset Niemann-Pick Disease, Type C

— $135 million equity financing with participation from leading specialist investors, corporate investors, and long-term growth partners, including Wellington Partners, JIC Venture Growth Investments (JIC VGI), Founders Fund, Narya Capital, Eisai Co., Ltd., and other select institutional investors —...

Rafael Holdings Announces Completion of Last Patient Last Visit for Pivotal Phase 3 TransportNPC™ Study Evaluating Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C

Topline results are expected to be announced in second half of calendar 2026 The company has had a pre-NDA meeting with the FDA and expects to submit an NDA in second half of calendar 2026 The company has received...

2025 Consensus Clinical Management Guidelines for Niemann–Pick Disease Type C

Published in the Journal of Inherited Metabolic Disease May 2026 INPDA is delighted to announce the publication of the 2025 Consensus Clinical Management Guidelines for Niemann–Pick Disease Type C in the Journal of Inherited Metabolic Disease. This important update...

Beren Therapeutics’ Adrabetadex NDA Accepted by FDA for Infantile-Onset Niemann-Pick Type C

Beren Therapeutics Announces FDA Acceptance of its New Drug Application for Adrabetadex in Infantile-Onset Niemann Pick Disease Type C Read this Clinical Trial update on the Beren Therapeutics website and learn more. February 23, 2026 • Accepted for priority...

NiemannPick India: Seen, Heard, Supported – Every Step Toward Diagnosis, Care & Hope