People aged 3 to under 8 who do not have deletions in exons 8 and/or 9 of the DMD gene and who test negative for anti-AAVrh74 antibodies are eligible to use ELEVIDYS. This is the first worldwide approval that covers people under the age of four.
Learn More: Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy
ELEVIDYS Gene Therapy Approved in Japan
Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases, today announced that the Japanese Ministry of Health, Labour, and Welfare (MHLW) has approved ELEVIDYS (delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy (DMD) under the conditional and time-limited approval pathway in Japan.
Read More: Will Elevidys Receive Approval from the European Medicines Agency (EMA)?
Exon 46-49 age 12 years
Nasıl faydanalanacağız bu durumdan sağlık bakanlığı aracılığıyla mı ne yapmamız gerekiyor
Türkiye Sağlık Bakanlığı’nın bu hususta bir şey yapabileceğini düşünmüyoruz.
Henüz Elevidys için Sarepta ya da Roche ile bir görüşmelerinin olmadığını düşünüyoruz.