New Treatment Modalities in Hemophilia
Küçük Resim Yok
Tarih
2022
Yazarlar
Dergi Başlığı
Dergi ISSN
Cilt Başlığı
Yayıncı
Erişim Hakkı
info:eu-repo/semantics/openAccess
Özet
Hemophilia is a single gene disorder and as a genetical coagulation system problem it is a life-long bleeding disorder. Even though routine treatment modalities as plasma-derived and then recombinant factor concentrates available for last 50 years, unmet needs is continuing for hemophilia therapy. Gold standart treatment is regularly prophylactic FVIII/FIX infusions. However, life-long and frequent intra-venous infusions become medical burden for patients and families. New agents as enhanced half-life (EHL) factor concentrates and non-factor therapies which are able to be used subcutaneously are very hopeful. In this review, EHL factor concentrates, FVIII mimetic agents and re-balancing therapies will be discussed. Although celluler gene therapy is very hopeful and successful phase-3 studies are reported, gene therapy for hemophilia will not be mentioned in this review.
Açıklama
Anahtar Kelimeler
Kaynak
Trends in pediatrics
WoS Q Değeri
Scopus Q Değeri
Cilt
3
Sayı
1
