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Found 5 clinical trials
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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 …

substrate reduction therapy
type 1 gaucher disease
replacement therapy
vpriv
velaglucerase alfa
  • 0 views
  • 22 May, 2021
  • 1 location
None
Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (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

  • 0 views
  • 24 Jan, 2021
  • 3 locations
None
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
None
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 …

  • 0 views
  • 29 Apr, 2021
  • 2 locations
None
A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies

The main aim of this study is to describe the safety profile of velaglucerase alfa (VPRIV) in participants with Gaucher disease type 1. Participants will be transitioning from other enzyme replacement therapies or substrate reduction therapies to VPRIV. Some participants may have already transitioned to treatment with VPRIV before this …

substrate reduction therapy
type 1 gaucher disease
replacement therapy
vpriv
velaglucerase alfa
  • 12 views
  • 22 Aug, 2021
  • 1 location