Joint Transnational Call (JTC2015)
- INSTINCT: Induced pluripotent stem cells for identification of novel drug combinations targeting cystic fibrosis lung and liver disease
- SMART-HaemoCare: Small Antibody Fragment as Alternative Tools in Haemophilia Care
- NSEuroNet : European network on Noonan syndrome and related disorders
- PERescue : Translating Peroxisome Biogenesis Disorders: Identifying Pharmacological Therapies and Clinical Trial Endpoints
- Propekal5: Tracing the untackled facets of Peeling Skin Disease-Targeting epidermal proteolysis for treatment
- EuroCID: Non-SCID combined immunodeficiencies: a diagnostic and therapeutic challenge
- iNSC-WMD: Patient-Derived Glial Precursor Cell Therapy for Vanishing White Matter Disease
- ImproveCPVT: Improving diagnosis and treatment of catecholaminergic polymorphic ventricular tachycardia: integrating clinical and basic science
- GENOMIT: Mitochondrial Disorders: from a genome-wide Registry to medical genomics, toward molecular mechanisms and new therapies
- INSAID: A comprehensive clinical and experimental approach to personalized molecular medicine in patients with defined and undefined autoinflammatory disorders
- EURO-CDG-2: A European research network directed towards improving diagnosis and treatment of inborn glycosylation disorders.
- ERAdicatPH: Understanding primary hyperoxaluria type 1 towards the development of innovative therapeutic strategies.
- KLKIN: Netherton Syndrome; From mechanism to therapies
- CoHEART: Improving Care for Cohesinopathies from heart phenotypes to novel therapies
- CMT-NRG: Modulation of Neuregulin signaling as an effective strategy to treat hereditary neuropathies (Charcot-Marie-Tooth disease)
- GETHERTHALPLUS: Novel Gene Therapy for Thalassemia: Pre-clinical Development and Assessment in Animal and Stem Cell Models
- PREPARE: Preparing for therapies in autosomal recessive ataxias
- Hipbi-RD: Harmonising phenomics information for a better interoperability in the RD field
- EuroDBA: The European Diamond-Blackfan Anemia Consortium