Orchard Therapeutics Announces Reimbursement Agreement in Spain [Yahoo! Finance]
KIRIN HLDGS CO LTD S/ADR (KNBWY)
US:NASDAQ Investor Relations:
kirinholdings.co.jp/english/ir
Company Research
Source: Yahoo! Finance
company, announced it has recently reached an agreement with the Interministerial Commission for the Pricing of Medicines of the Directorate of Common Portfolio of Services of the Spanish National Health System (SNS) which will result in reimbursed access to Libmeldy® (atidarsagene autotemcel) for all eligible children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ)—collectively referred to as early-onset—metachromatic leukodystrophy (MLD). MLD is an ultra-rare, rapidly progressive, irreversible and ultimately fatal neurometabolic disease caused by an error in the gene responsible for encoding the enzyme arylsulfatase A (ARSA) leading to neurological damage and developmental regression. In the most severe form of MLD, babies develop normally but in late infancy start to rapidly lose the ability to walk, talk and interact with the world around them. These children eventually deteriorate into a vegetative sta
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