Friends of rare disease research

Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1)

Personalized Training for People With Rare Neuromuscular Disorders

120 | Oslo University Hospital

Headache Disorders

RegistRare: a Retro-prospective Registry of Rare Primary Headaches in Italian T...

3000 | Silvia Benemei

Rare Diseases of Genetic Origin, Rare Forms of Common Diseases Suspected of Being Genetic in Origin

Identification of the Molecular and/or Pathophysiological Bases of Rare Disease...

850 | Dijon University Hospital Center

Prader-Willi Syndrome, PWS-like Syndrome, Silver Russel Syndrome, Congenital Hypopituitarism, Klinefelter (XXY-)Syndrome, Congenital Adrenal Hyperplasia, XXXXY Syndrome, XXYY Syndrome, XXXX Syndrome (Tetra-X Syndrome), Disorders of Sex Development, Turner Syndrome, 46, XY DSD, Tuberous Sclerosis, Neurofibromatosis, Albright Hereditaire Osteodystrofie, Cornelia de Lange Syndrome, Saethre-Chotzen Syndrome, 17p- Deletiesyndrome, VCF Syndrome, POLR3A Mutatie, Ohdo Syndrome, Jacobsen Syndrome / 11 q Syndrome, Myrhe Syndrome, CHARGE Syndrome, 1q25-32 Deletie, Bardet Biedl Syndrome, Rett Syndrome, 22q11 Deletion Syndrome, Allan-Herndon-Dudley Syndrome, Kallmann Syndrome, Rare Bone Disorders, Noonan Syndrome, Williams-Beuren Syndrome

GROWing Up With Rare GENEtic Syndromes

600 | Dr. Laura C. G. de Graaff-Herder

Rare Diseases

A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.

8 | Swedish Orphan Biovitrum

Sleep Disorders, Circadian Rhythm, Cognitive Dysfunction

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

40 | Max Zeller Soehne AG

Pulmonary Arterial Hypertension

Safety and Efficacy of BIA 5-1058 in PAH

20 | Bial - Portela C S.A.

Fournier Gangrene

Fournier's Gangrene in Patient With Uncontrolled Type 2 Diabetes

1 | University of Catania