Study of SHP620 (Maribavir) in Healthy Adults The purpose of this study is to determine how an investigational treatment (maribavir) is handled by the body when administered with two already approved drugs (digoxin and dextromethorpha...

• Country None
• organs None
• Specialty None

Home Therapy With VPRIV in Gaucher's Disease The purpose of this study is to proof increasing patient satisfaction and preservation of quality of life in patients with Gaucher's Disease receiving their enzyme replacement therapy with...

• Country None
• organs None
• Specialty None

Observational Natural History Study of Autosomal Dominant Retinitis Pigmentosa (adRP) The purpose of this study is to gain an understanding of how adRP progresses over time in patients with misfolded rod opsin mutations.

• Country None
• organs None
• Specialty None

A Comparison of Lanthanum Carbonate and Calcium Carbonate in Children To provide information about the safety and efficacy of lanthanum carbonate in children compared to standard treatment.

• Country Argentina, Bulgaria, Chile, Czech Republic, Germany, Hungary, Poland, Romania, Russian Federation, South Africa, Turkey
• organs None
• Specialty None

Long-term Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome Who Completed the TED-C13-003 Study The purpose of this study is to evaluate the safety and efficacy of teduglutide treatment of children with short bowel syndrome (SBS) who completed the TED-C13-003 study over a long-term p...

• Country None
• organs None
• Specialty None

A Multicenter Study to Evaluate the Efficacy and Safety of Cinryze® for the Treatment of Acute Antibody-mediated Rejection in Patients With Kidney Transplant To evaluate the efficacy of Cinryze® given for the treatment of acute antibody-mediated rejection (of renal allograft) (AMR) in kidney transplant recipients as measured by the proportion o...

• Country Canada, France, Germany, Netherlands, Spain, United States
• organs None
• Specialty None

THIN Database Study: Resource Use and Outcomes in Patients With Adrenal Insufficiency Prescribed Hydrocortisone: Immediate, or Modified Release, or Prednisolone To describe clinical outcomes and both primary and secondary care health care resource use in patients who have been prescribed either immediate-release (IR) hydrocortisone or modified-rel...

• Country None
• organs None
• Specialty None