Cereno Scientific , a company developing innovative treatments for common and rare cardiovascular disease, today announced that the company has been granted approval by the FDA for Expanded Access,.
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02 marzo 2021 17:14
Fonte: Adnkronos
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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.