The page lists 60 projects related to the topic "orphan".
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1 | DE-ORPHAN | DEtermination of Orphan Receptor PHysiological Agonists and sigNals | 2015 |
2 | RETHRIM | Restoring tissue regeneration in patients with visceral Graft versus Host Disease. | 2015 |
3 | SC0806 | Regenerative treatment of complete Traumatic Spinal Cord injury with a surgical implantation of a biodegradable device with FGF1 and nerve grafts | 2015 |
4 | ROR2BONE | Investigating Ror2-dependent non-canonical Wnt signaling in bone remodeling | 2015 |
5 | FraxiFam | Reconstructing gene family evolution in the ash genus (Fraxinus | 2015 |
6 | DEALS | Biomarker assay development to expedite ALS experimental therapeutic advancement | 2015 |
7 | GLIOMARK | Validation of blood-brain-barrier permeability as a glioma biomarker by means of the radiotracer 99mTc-tetrofosmin and single-photon emission computer tomography | 2015 |
8 | PRION2020 | Function and malfunction of the prion protein | 2015 |
9 | TETRA | Autologous Stem Cell Seeded Tissue Engineered Trachea | 2016 |
10 | FAST-DEVELOPS | Developing new therapeutics for Friedreich ataxia | 2015 |
11 | SCIDNET | DevelopIng Genetic medicines for Severe Combined Immunodeficiency (SCID) | 2016 |
12 | VISION DMD | VISION-DMD - Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy | 2016 |
13 | MYOCURE | Development of an innovative gene therapy platform to cure rare hereditary muscle disorders | 2016 |
14 | H2020MM04 | DC-based immunotherapy to treat Malignant Mesothelioma | 2016 |
15 | AML-VACCiN | Clinical development of a dendritic-cell vaccine therapy for acute myeloid leukaemia | 2016 |
16 | WISLAS | Warmed Icelandic Soils, Lipids and Sequencing: towards a better understood climate proxy | 2016 |
17 | RiboDisc | Discovery of novel orphan riboswitch ligands | 2016 |
18 | BCM-UPS | Dissecting the role of the ubiquitin proteasome system in the pathogenesis and therapy of B-cell malignancies | 2016 |
19 | EMODI | Epithelial resistance modulation to treat disease | 2016 |
20 | NOTID | Novel treatment of inflammatory diseases | 2016 |
21 | OVOMATIC | IN-OVO AUTOMATION FOR CANCER DRUG DISCOVERY | 2017 |
22 | CureTheCloves | PIK3CA inhibition in patients with the CLOVES syndrome | 2017 |
23 | CoALIVE | Coenzyme A – Launch of In Vivo Experiments | 2017 |
24 | GeneVision | Developing a cure for retinitis pigmentosa due to Usher syndrome | 2017 |
25 | Genome Biologics | Reducing the cost and increasing the speed of preclinical research in the pharmaceutical industry | 2017 |
26 | CISTEM | Heart On chip based on induced pluripotent Stem cell Technology for personalized Medicine | 2018 |
27 | TERRESTREVOL | Exploring terra incognita: terrestrialization of arthropods in the era of genomics | 2018 |
28 | UshTher | Clinical trial of gene therapy with dual AAV vectors for retinitis pigmentosa in patients with Usher syndrome type IB | 2018 |
29 | MCDS-Therapy | Repurposing of carbamazepine for treatment of skeletal dysplasia | 2017 |
30 | RECOMB | Stem-cell based gene therapy for recombination deficient SCID (RECOMB) | 2018 |
31 | CLINGLIO | A Clinical Phase IIB trial with 2OHOA in patients with newly-diagnosed malignant glioma. | 2017 |
32 | OligoGpivotalCF | A pivotal phase IIb clinical trial of inhaled alginate oligosaccharide (OligoG) for cystic fibrosis | 2018 |
33 | AirBorne | AerIal RoBotic technologies for professiOnal seaRch aNd rescuE | 2018 |
34 | EDROMA | Novel nanomedicine for the treatment of glioma | 2018 |
35 | CodingHeart | Novel Coding Factors in Heart Disease | 2019 |
36 | CARAMBA | SLAMF7-CAR T cells prepared by Sleeping Beauty gene-transfer for immunotherapy of multiple myeloma – a rare hematologic disease | 2018 |
37 | SMARTRIOX | Disruptive targeted drug delivery system via synergistic combination of intelligent DNA molecular machines and gated mesoporous nanoparticles | 2018 |
38 | PAH-HOPE | New hope for the PAH patient: A Novel Therapeutic for Pulmonary Arterial Hypertension (PAH) | 2018 |
39 | GENBIO | Reducing the cost, time, and ethical burden of preclinical research in the pharmaceutical industry | 2018 |
40 | KHON2bTREAT | Late Clinical Development of KH176: an innovative orphan drug to reach mitochondrial disease patients & market | 2018 |
41 | MIN4ALD | Use of MIN-102 for the treatment of children with cerebral ALD | 2018 |
42 | denovoSkin | Personalized, bio-engineered skin grafts for the permanent treatment of skin defects. | 2018 |
43 | NeuroProtect | Novel Drug Therapy with Potential to Cure Neuro-Degenerative Diseases | 2018 |
44 | denovoSkin | Personalized, bio-engineered skin grafts for the permanent treatment of skin defects | 2018 |
45 | Treat-HSP | Development of new therapy for rare motor neuron diseases | 2018 |
46 | BERMES | A Novel BERberine derivative for Malignant MESothelioma | 2018 |
47 | iProg | Tailored cell-based therapies for frontotemporal dementia and related genetically defined CNS orphan indications | 2018 |
48 | TOL2 | An effective and safe targeted therapy for treatment of Myasthenia gravis | 2018 |
49 | deCrYPtion | Decrypting Mycobacterium cytochrome P450 (CYP) physiological functions by testing hypotheses emitted form large-scale comparative genomics analysis | 2019 |
50 | SUPERTEFF | A new paradigm in boosting orphan crops to super grains: Linking metabolomics and gene editing to improve Teff for global food security and sustainable agriculture | 2020 |
51 | Hyalcis | Hyaluronic-Cisplatin Film for Implant in Malignant Mesothelioma Recurrence | 2019 |
52 | RAPID | The first effective remedy for acute pancreatitis disease | 2019 |
53 | Chelator | IRON CHELATOR NBMI FOR THE TREATMENT OF IRON OVERLOAD IN BETA-THALASSEMIA MAJOR | 2019 |
54 | SPELITEC | Sequence sPecific ELImination of shiga-Toxin E. Coli | 2019 |
55 | 3TR | Identification of the Molecular Mechanisms of non-response to Treatments, Relapses and Remission in Autoimmune, Inflammatory, and Allergic Conditions | 2019 |
56 | TRS | A paradigm change in the treatment of blinding ocular diseases | 2019 |
57 | MaxONCO | A cancer vaccine to boost body’s natural defences to fight chordoma and head and neck cancer | 2020 |
58 | NeuroPa | Targeting the brain sigma-1 receptor: A paradigm shift for the treatment of neuro-degenerative disorders | 2019 |
59 | RD-MRI NETWORK | BRIDGING THE GAP BETWEEN ACADEMIA & CLINICAL TRIALS FOR THE DEVELOPMENT OF RARE DISEASE TREATMENTS VIA NUCLEAR MAGNETIC RESONANCE IMAGING (MRI) | 2020 |
60 | ERA | Epigenetic Regulation in Acinetobacter baumannii | 2020 |