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Found 4 clinical trials
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

The study will provide information on outcomes in people with type 1 Gaucher disease when they are treated with velaglucarase alfa (also called VPRIV), under standard care. Standard care means the participant will be treated according to the clinic's standard practice. The study sponsor will not be involved in how …

replacement therapy
vpriv
velaglucerase alfa
type 1 gaucher disease
substrate reduction therapy
  • 0 views
  • 22 May, 2021
  • 1 location
Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (EMTISTD)

management of respiratory problems. Recent studies have suggested new methods of treatment, such as enzyme replacement therapy, bone marrow transplantation and substrate reduction therapy. The first

  • 36 views
  • 19 Apr, 2022
  • 3 locations
A Long-term Follow-up Study of Gaucher Disease

The study aims are to: a) identify the long-term natural history of Gaucher disease, b) evaluate long-term treatment efficacy of enzyme replacement therapy (ERT) and substrate reduction therapy

replacement therapy
substrate reduction therapy
  • 26 views
  • 30 Sep, 2021
  • 1 location
World Data on Ambroxol for Patients With GD and GBA Related PD

Ambroxol hydrochloride is an oral mucolytic drug available over-the-counter for many years as cough medicine. In 2009 it was found to also act as a pharmacological chaperone (PC) for mutant glucocerebrosidase, albeit in a several-fold higher dose. Unfortunately, due to its low cost, there have been no pharma-driven clinical trials …

type 1 gaucher disease
alglucerase
  • 0 views
  • 25 Jan, 2022
  • 2 locations