Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS)

Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS)

Limited real-world data from routine clinical care are available on the safety and effectiveness of treatment with taliglucerase alfa in patients with Gaucher disease (GD). Taliglucerase Alfa Surveillance (TALIAS), a multinational prospective Drug Registry of patients with GD, was established to eva...

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Veröffentlicht in:Orphanet journal of rare diseases 2022-04, Vol.17 (1), p.145-145, Article 145
Hauptverfasser: Titievsky, Lina, Schuster, Tilman, Wang, Ronnie, Younus, Muhammad, Palladino, Andrew, Quazi, Kabir, Wajnrajch, Michael P, Hernandez, Betina, Becker, Pamela S, Weinreb, Neal J, Chambers, Christina, Mansfield, Roy, Taylor, Louise, Tseng, Li-Jung, Kaplan, Paige
Format: Artikel
Sprache:eng
Schlagworte:
Adolescent
Adults
Back pain
Blood cell count
Child
Clinical trials
Drug therapy
Effectiveness
Enzyme Replacement Therapy - adverse effects
Enzymes
FDA approval
Female
Gaucher disease
Gaucher Disease - drug therapy
Gaucher's disease
Glucosylceramidase - adverse effects
Hemoglobin
Humans
Lupus
Male
Medical research
Non-interventional study
Patients
Pediatrics
Platelets
Rare diseases
Registries
Safety
Spleen
Statistics
Taliglucerase alfa
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Zusammenfassung:Limited real-world data from routine clinical care are available on the safety and effectiveness of treatment with taliglucerase alfa in patients with Gaucher disease (GD). Taliglucerase Alfa Surveillance (TALIAS), a multinational prospective Drug Registry of patients with GD, was established to evaluate the long-term safety (primary objective) and effectiveness (secondary objective) of taliglucerase alfa. We present an interim analysis of the data from the Drug Registry collected over the 5-year period from September 2013 to January 2019. A total of 106 patients with GD (15.1% children aged 
ISSN:1750-1172
1750-1172
DOI:10.1186/s13023-022-02289-7