A study to assess the renal function in young male patients with Fabry disease who have never received any specific treatment for this disease. Estudio para evaluar la función renal en varones jóvenes con enfermedad de Fabry los cuales no han recibido nunca ningún tratamiento específico To document renal function and other Fabry disease manifestations across age in treatment-naïve, young male patients with Fabry disease. Documentar la función renal y otras manifestaciones de la...

• Pays Aucun
• Organes Aucun
• Spécialités Aucune

A trial of antigen-specific immune tolerance induction in mucopolysaccharidosis I (MPS I) patients initiating enzyme replacement therapy with Aldurazyme® (laronidase) To evaluate the antibody response to Aldurazyme in newly treated severe MPS I patients following an antigen-specific immunosuppressive regimen.

• Pays Aucun
• Organes Aucun
• Spécialités Aucune