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02 marzo 2021 17:14 Fonte: Adnkronos #salute-e-benessere AUSTIN, Texas, March 2, 2021 /PRNewswire/  Beyond Batten Disease Foundation (BBDF) is pleased to announce an agreement with Actelion Pharmaceuticals Ltd, a Janssen Pharmaceutical Company of Johnson & Johnson, to provide Zavesca® (miglustat) for the development of BBDF-101, a proprietary combination of miglustat and trehalose to treat juvenile Batten disease (CLN3). Janssen will provide drug supply for the clinical trials and Extended Access Program, and right of reference to data within the Zavesca New Drug Application (NDA) to support the development of BBDF-101. CLN3, one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), is a rare, fatal, inherited, neurodegenerative disease for which there is no treatment. Since it was founded in 2008, BBDF has spearheaded the development of BBDF-101.

Nordrhein westfalenUnited statesForbes porterMary beth kiserCraig bensonAngela schulzErika augustineHuman developmentBatten disease foundationbeyond foundationAdvisory committeeUniversity medical center hamburgActelion pharmaceuticals ltdBatten disease foundationJanssen pharmaceutical company of johnsonUniversity of rochesterAccess program