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BioMarin stopped providing the drug for free at the beginning of the year after negotiations with Belgian health authorities regarding reimbursement of the product repeatedly failed. This caused the parents to start legal proceedings to force the company to keep providing the medicine free of charge. BioMarin was ordered to keep doing so until ...
Prosensa was then acquired by BioMarin Pharmaceutical, [7] which continued the development of drisapersen [8] and was concurrently working with similar exon skipping therapies for other exons. [9] Both drisapersen and the similar drug eteplirsen have filed a New Drug Application (NDA) for review with the US Food and Drug Administration (FDA). [10]
BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities in the United States , South America , Asia , and Europe . BioMarin's core business and research is in enzyme replacement therapies (ERTs).
The DOJ has asked the company to submit certain documents related to the sponsored testing of its therapies Vimizim and Naglazyme, BioMarin said. "We have produced documents in response to the ...
Amifampridine phosphate is used as a drug, predominantly in the treatment of a number of rare muscle diseases. The free base form of the drug has been used to treat congenital myasthenic syndromes and approved by the FDA for Lambert–Eaton myasthenic syndrome (LEMS) through compassionate use programs since the 1990s and was recommended as a first line treatment for LEMS in 2006, using ad hoc ...
Ringaskiddy (Irish: Rinn an Scidígh, meaning 'Skiddy's Headland') [2] is a village in County Cork, Ireland. It is located on the western side of Cork Harbour , south of Cobh , and is 15 kilometres (9 mi) from Cork city, to which it is connected by the N28 road .
Martin Shkreli (/ ˈ ʃ k r ɛ l i /; born March 17, 1983) is an American investor and businessman.Shkreli is the co-founder of the hedge funds Elea Capital, MSMB Capital Management, and MSMB Healthcare, the co-founder and former CEO of pharmaceutical firms Retrophin and Turing Pharmaceuticals, and the former CEO of start-up software company Gödel Systems, which he founded in August 2016.
Vosoritide was developed by BioMarin Pharmaceutical and got orphan drug status in the US as well as the European Union. [ 5 ] [ 7 ] [ 13 ] [ 9 ] The safety and efficacy of vosoritide in improving growth were evaluated in a year-long, double-blind, placebo-controlled, phase III study in participants five years and older with achondroplasia who ...