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Global Diagnostics Partnering Deals Report/Directory 2021: Trends, Players and Financials 2014-2021

Global Diagnostics Partnering Deals Report/Directory 2021: Trends, Players and Financials 2014-2021
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Global Genetic Disorders Partnering Deals Report/Directory 2021: Trends, Players and Financials 2014-2021

Global Genetic Disorders Partnering Deals Report/Directory 2021: Trends, Players and Financials 2014-2021
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Global Genetic Disorders Partnering Deals Report/Directory 2021: Trends, Players and Financials 2014-2021

Global Genetic Disorders Partnering Deals Report/Directory 2021: Trends, Players and Financials 2014-2021
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'Star Wars' star Mark Hamill's extraordinary visit with a dying boy

'Star Wars' star Mark Hamill's extraordinary visit with a dying boy
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Beyond Batten Disease Foundation Announces Agreement with Actelion [...]

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 Westfalen , United States , Forbes Porter , Mary Beth Kiser , Craig Benson , Angela Schulz , Erika Augustine , Human Development , Batten Disease Foundationbeyond Foundation , Advisory Committee , University Medical Center Hamburg , Actelion Pharmaceuticals Ltd , Batten Disease Foundation , Janssen Pharmaceutical Company Of Johnson , University Of Rochester , Access Program , Us Federal Drug Administration , Beyond Batten Disease Foundation , Janssen Pharmaceutical Company , Extended Access Program , Zavesca New Drug Application , Generally Regarded , Drug Administration , Texas Children , Eunice Kennedy Shriver National Institute , Child Health ,