Rare Diseases

A large group of diseases which are characterized by a low prevalence in the population. They frequently are associated with problems in diagnosis and treatment.

Drugs by status

Phase 4 trials (1)

burosumab-twza

Phase 3 trials (4)

irinotecan
Filgrastim
temozolomide
modafinil

Phase 2 trials (5)

crofelemer
durvalumab
cannabidiol
tremelimumab
a166

Phase 1 trials (2)

pbi-shrna ews/fli1 type 1 lpx
emapalumab-lzsg

Other trials (1)

fluoxetine

Organizations

Organization Involved with Phase 4 Indications (1)

Assistance Publique - Hôpitaux de Paris

Organization Involved with Phase 3 Indications (2)

Gradalis, Inc.
University of Lyon

Organization Involved with Phase 2 Indications (8)

AstraZeneca
MedImmune
Novartis
Greenville Health System
Oregon Health and Science University
Seoul National University
University of Texas at Houston
Klus Pharma Inc.

Organization Involved with Phase 1 Indications (1)

Strike Bio, Inc.

Organization Involved with Other Experimental Indications (1)

Stealth BioTherapeutics Inc.