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Cereno Scientific is granted Expanded Access by the FDA to drug candidate CS1 for rare disease pulmonary arterial hypertension

Ascendis Pharma A/S (NASDAQ:ASND) Q1 2023 Earnings Call Transcript

Operator: Thank you for standing by and welcome to the Q1 2023 Ascendis Pharma Earnings Conference Call. At this time, all participants are in a listen-only mode.

Gamida Cell Reports Full Year 2022 Financial Results and Provides Company Update

Company reports on productive interactions with FDA and preparations for launch of omidubicel, if approved, in advance of May 1 PDUFA date Company outlines strategic restructuring, plans to. | March 27, 2023

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.

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