International Niemann-Pick Disease Alliance (INPDA)

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...
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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...
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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...
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