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Morbus Farby neue Therapien



Short title: Migalastat Beob. Stu

Investigated disease: Morbus Fabry

Principal Investigator: Dr. Sima Canaan-Kühl


Clinic, Institute: Medizinische Klinik m.S. Nephrologie und Internistische Intensivmedizin CCM

Patient (diseased)
Age: 18 - 99
Gender: All

Status: Participants wanted
Last change: 22.09.2021

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Study Description

Title

Morbus Farby neue Therapien

Short title

Migalastat Beob. Stu

General Short Description

The objective of the study is to generate long term data on treatment with Migalastat under "real world" conditions. The selection of patients will be based on the SmPC/Fachinformation. The treatment duration/patient will be 2 years. All patients will be followed-up by the German Fabry expert centers that are listed below.

Scientific Short Description

German observational multicenter study of patients with Fabry disease under chaperone therapy with Migalastat-HCl.

Investigated Disease, Health Issue

ICD-Code
  • E75.2 - Sonstige Sphingolipidosen

Inclusion Criteria

Age

18 - 99

Gender

All

Additional Inclusion Criteria

The objective of the study is to generate long term data on treatment with Migalastat under "real world" conditions. The selection of patients will be based on the SmPC/Fachinformation. The treatment duration/patient will be 2 years. All patients will be followed-up by the German Fabry expert centers that are listed below.

Exclusion Criteria

The objective of the study is to generate long term data on treatment with Migalastat under "real world" conditions. The selection of patients will be based on the SmPC/Fachinformation. The treatment duration/patient will be 2 years. All patients will be followed-up by the German Fabry expert centers that are listed below.

Characteristics

Studytype

Non-interventional

Studyphase

N/A

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