In 2012, BioMarin began the first clinical trial on affected patients using their recombinant DNA technology cerliponase alfa which is synthesized using Chinese hamster ovarian (CHO) cell lines. After the success of this clinical trial, the U.S. FDA approved the marketing of cerliponase alfa to patients with CLN2 disease. The approval only applied to patients three years or older as the FDA wants to have more data available on children under the age of three before approving it for younger patients. A ten-year study is being performed to assess the long term effects of continued use of this drug. Cerliponase alfa is developed by BioMarin Pharmaceutical and the drug application was granted both orphan drug designation to provide incentives for rare disease research and the tenth Rare Pediatric Disease Priority Review Voucher. Cerliponase alfa was also approved by European Medicines Agency (EMA) on 30 May 2017. In the United Kingdom NICE evaluated cerliponase alfa for the treatment of CLN2 and deemed it not cost-effective. BioMarin announced that the price per infusion is $27,000, coming to $702,000 per year for treatment, though using Medicaid can decrease the cost.In March 2018, cerliponase alfa was approved in the United States as a treatment for a specific form of Batten disease.
Amédée Galzin (1 May 1853, Parrinet, Aveyron – 14 February 1925, Parrinet) was a French veterinarian and mycologist. In 1878 he obtained his degree from the veterinary college in Toulouse. From 1879 to 1905, he served as a military veterinarian, becoming a knight of the Legion of Honour in 1899.With Abbé Hubert Bourdot, he was co-author of a series of publications (11 parts, 1909 to 1925) involving Hymenomycetes native to France; all parts being published in the Bulletin de la Société Mycologique de France. With Bourdot, he also wrote Heterobasidiae nondum descriptae (Descriptions of a few jelly fungi).With Bourdot, he was the taxonomic authority of the fungi genus Oxyporus, as well as of numerous mycological species. == References ==