Taliglucerase alfa in the longterm treatment of children and adolescents with type 1 Gaucher disease: the Albanian experience

• Hoofdauteurs: Paskal Cullufi (Auteur), Sonila Tomori (Auteur), Virtut Velmishi (Auteur), Agim Gjikopulli (Auteur), Ilir Akshija (Auteur), Aferdita Tako (Auteur), Ermira Dervishi (Auteur), Gladiola Hoxha (Auteur), Marjeta Tanka (Auteur), Erjon Troja (Auteur), Mirela Tabaku (Auteur)
• Formaat: Boek
• Gepubliceerd in: Frontiers Media S.A., 2024-02-01T00:00:00Z.
• Onderwerpen: article
• Online toegang: Connect to this object online.

IntroductionEnzyme replacement therapy is already recognized as the gold standard of care for patients with Gaucher disease. Taliglucerase alfa is one of the three alternatives recommended for treatment of Gaucher disease in children and adults.AimThis study aims to evaluate the long-term efficacy a...