Friends of rare disease research

Paraganglioma/ Phaeochromocytoma, Melanoma and Other Malignant Neoplasms of Skin, Gastrointestinal Stromal Tumor (GIST), Adrenocortical Tumor, Pancreatic Tumors, Esthesioneuroblastoma, Olfactory, Mesothelioma, Nasopharyngeal Carcinoma (NPC), NUT Carcinoma, Pleuropulmonary Blastoma, Salivary Gland Tumors, Thymic Tumors, Thyroid Carcinoma, Appendiceal Cancers

An International Study on Pediatric Patients With Rare Tumors.

6250 | Padua Hospital

Non-small Cell Lung Cancer

Survival Outcome of EGFR-TKI in Uncommon EGFR Mutant Advanced NSCLC

800 | Hunan Province Tumor Hospital

Polycythemia Vera, ET (Essential Thrombocythemia), Polycythemia Vera (PV), Essential Thrombocythemia (ET), Primary Myelofibrosis (MF), Primary Myelofibrosis (PMF), Myelofibrosis, Myelofibrosis (MF), Myelofibrosis, Primary, Myelofibrosis, Post ET, Myelofibrosis, Post PV, Myelofibrosis (PMF), Myelofibrosis，MF, Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis, Myelofibrosis Due to and Following Polycythemia Vera, Myelofibrosis Transformation in Essential Thrombocythemia, Myelofibrosis With High Molecular Risk Mutations, MF, Secondary Myelofibrosis, Secondary Myelofibrosis in Myeloproliferative Disease, Secondary Myelofibrosis (Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis), Post-Polycythemia Vera Myelofibrosis, Post-polycythemia Vera Myelofibrosis (PPV-MF), Post-polycythemia Vera Myelofibrosis (Post-PV MF), Post-polycythemia Vera Myelofibrosis(Post-PV MF), Post-PV MF, Post-Essential Thrombocythemia Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis (PET-MF), Post-essential Thrombocythemia Myelofibrosis(Post-ET MF), Post-essential Thrombocythemia Myelofibrosis (Post-ET MF), Post-ET MF, Pre-fibrotic Myelofibrosis, Myeloproliferative Disorder, Myeloproliferative Disorders, Myeloproliferative Disorders (MPD), Myeloproliferative Neoplasms (MPNs), Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis, Myeloproliferative Neoplasm With 10% Blasts or Higher, Myeloproliferative Neoplasms, MPN, MPN (Myeloproliferative Neoplasms), MPN-associated Myelofibrosis, Myeloproliferative Neoplasm, Unclassifiable, Myeloproliferative Neoplasm, Not Otherwise Specified, Accelerated Phase MPN, Accelerated Phase Myeloproliferative Neoplasm, Blast Phase MPN, Blast Phase Myeloproliferative Neoplasm, Thrombocythemia Myelofibrosis (PET-MF), Thrombocythemia, Essential, Thrombocythemia, Hemorrhagic, Agnogenic Myeloid Metaplasia, Chronic Idiopathic Myelofibrosis, Idiopathic Myelofibrosis, MDS/MPN Crossover Syndromes

MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, an...

5000 | MPN Research Foundation

Pulmonary Alveolar Proteinosis

A National Registry For Pulmonary Alveolar Proteinosis

500 | Children's Hospital Medical Center, Cincinnati

Metachromatic leukodystrophy

The Natural History of Metachromatic Leukodystrophy Study (HOME Study)

21 | National Organization for Rare Disorders

Gastrointestinal Cancer, Neuroendocrine Tumours, Malignant Female Reproductive System Neoplasm

A Phase II Trial of Ipilimumab and Nivolumab for the Treatment of Rare Cancers

120 | Olivia Newton-John Cancer Research Institute

Periodic Paralyses

Utility and Validation's Study of a Smartphone Application for Periodic Paralys...

60 | National Institute of Health and Medical Research, France

Sleep Disorders, Circadian Rhythm, Cognitive Dysfunction

Feasibility Study on the Use of Redormin®500 on Day-time Cognition

40 | Max Zeller Soehne AG