GAIN
German network for multi-organ autoimmune diseases
Multi-organ autoimmune diseases belong to the “ultra-rare” disorders and have only recently been recognized being caused by monogenetic mutations in immune-regulatory genes. Patients are typically characterized by multi-organ inflammation
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TreatHSP.net
Translational research on Hereditary Spastic Paraplegias
Hereditary Spastic Paraplegias (HSP) are clinically and genetically highly heterogeneous neurodegenerative disorders primarily affecting the long axons of the corticospinal tract, leading to progressive lower limb spasticity and weakness.
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ADDRess
Translational Research for Persons with Abnormal DNA Damage Response
Disorders with Abnormal DNA Damage Response (DADDR) are rare genetic conditions with impaired DNA repair or maintenance. A shared feature among affected individuals is a strongly increased cancer risk. Treatment is commonly complicated due to the underlying genetic defect.
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STOP-FSGS
Speed Translation-Oriented Progress to Treat FSGS
Idiopathic (primary) FSGS is a rare disease affecting an estimated 2,000 people in Germany. Its secondary form represents a major cause for irreversible loss of renal function and life-long need for dialysis or transplantation.
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CONNECT-GENERATE
German Network for Research on Autoimmune Encephalitis
The research network CONNECT-GENERATE aims at improving the diagnosis and treatment of people with autoimmune encephalitis, a rare but treatable form of brain inflammation.
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Treat-ION
New Therapies for Neurological Ion Channel and Transporter Disorders
Treat-ION represents a network of clinicians and scientists across Germany to advance the knowledge about recognizing and treating rare neurological ion channel and transporter disorders. Those comprise a variety of neuropsychiatric diseases and symptoms including developmental delay, epilepsy, episodic and chronic ataxia, migraine and others.
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MyPred
Network for young individuals with syndromes predisposing to myeloid malignancies
During the last decade, an increasing number of novel genetic disorders with predisposition to myeloid malignancy in young individuals has been identified. Like the inherited bone marrow failure syndromes, some of these disorders are associated with other organ dysfunction.
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HiChol
Translational network for Hereditary Intrahepatic Cholestasis
Defective bile formation is referred to as cholestasis and can be caused by mutations in genes important for bile formation in the liver. Cholestasis as result of genetic variants is referred to as hereditary intrahepatic cholestasis and comprises a heterogeneous group of rare disorders.
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NEOCYST
Network of Early Onset Cystic Kidney Disease
Hereditary cystic kidney diseases are among the most common causes of chronic renal failure in children. As a multidisciplinary network of clinicians, geneticists and basic scientific researchers, NEOCYST is dedicated to the comprehensive research and improved care of patients.
continueTranslational research on rare diseases
Many of the approximately 8,000 rare diseases are still poorly understood. To address this, the Federal Ministry for Research, Technology, and Space (BMFTR) funded cross-site research projects within translational research networks from 2003 through the end of 2026. The core objective remains unchanged: to improve diagnostics and fast-track promising scientific discoveries into clinical practice, ultimately enhancing healthcare and quality of life for individuals living with rare diseases.
Even after the official funding period has concluded, the research networks continue working toward their goals, depending on available resources and infrastructure. The content under the “Research Networks” section will remain actively updated, particularly regarding scientific publications. Please note, however, that some of the listed projects may now operate on a reduced scale or have already been successfully completed.
The Translational Science of Rare Diseases – From Rare to Care V
In April 2026, the German research networks for rare diseases once again brought together leading experts at the international symposium The Translational Science of Rare Diseases – From Rare to Care V to discuss the latest breakthroughs in the field. The event centered on innovative approaches to fast-tracking scientific discoveries into concrete therapies and enhancing care for patients living with rare diseases.
Programme booklet: The Translational Science of Rare Diseases – From Rare to Care V – 2026