Metastatic glioma
no clinical trials 5 substances studiedView
Rare diseases archive
Explore rare diseases studied in clinical trials – page 16
619 rare diseases in the catalogue0 linked trials
Top 10 most studied rare diseases
Rare diseases with the highest number of active trials right now.
No. 1 most studied
Acoustic neuroma
0Active trials · 1% of the top 10
- recruiting now
- 0
- countries
- 0
- open to children
- 0
- substance tested
- 1
Shares are of these ten diseases' active trials together.
All rare diseases
Browse every rare disease currently studied across European trials.
A–Z
619 rare diseases
Metastatic renal cell carcinoma
no clinical trials 11 substances studiedView
- Substances:AxitinibCabozantinib+9 more
Mitochondrial DNA depletion
no clinical trials 2 substances studiedView
- Substances:DoxecitineDoxribtimine
Mitochondrial myopathy
no clinical trials 4 substances studiedView
- Substances:DoxecitineDoxribtimine+2 more
Mixed-type liposarcoma
no clinical trials 2 substances studiedView
- Substances:TrabectedinTtf-Ngr
Monogenic diabetes
no clinical trials 4 substances studiedView
- Substances:EmpagliflozinGlimepiride+2 more
Mucopolysaccharidosis
no clinical trials 11 substances studiedView
- Substances:BusulfanIdursulfase+9 more
Mucopolysaccharidosis II
no clinical trials 3 substances studiedView
- Substances:IdursulfasePabinafusp Alfa+1 more
Mucopolysaccharidosis III
no clinical trials 2 substances studiedView
Mucous membrane pemphigoid
no clinical trials 2 substances studiedView
- Substances:AldesleukinLactose
Multifocal motor neuropathy
no clinical trials 3 substances studiedView
- Substances:Dnth103Empasiprubart+1 more
Multiple system atrophy
no clinical trials 5 substances studiedView
- Substances:AMLENETUGEmrusolmin+3 more
Muscular dystrophy
no clinical trials 8 substances studiedView
- Substances:Abiraterone AcetateDeflazacort+6 more
Myasthenia gravis
no clinical trials 39 substances studiedView
- Substances:AUR200Batoclimab+37 more
Mycobacterium abscessus infection
no clinical trials 16 substances studiedView
- Substances:AmikacinAzithromycin+14 more
Mycobacterium avium complex infection
no clinical trials 3 substances studiedView
- Substances:AzithromycinClarithromycin+1 more
Myelodysplastic syndrome
no clinical trials 72 substances studiedView
- Substances:Ag-946Ag-946 Phosphate+70 more
Myelodysplastic syndrome transformation
no clinical trials 5 substances studiedView
- Substances:AllopurinolRasburicase+3 more
Myelodysplastic syndrome with excess blasts
no clinical trials 2 substances studiedView
- Substances:GilteritinibMidostaurin
Myelofibrosis
no clinical trials 38 substances studiedView
- Substances:ALKYLATING AGENTSANTIMETABOLITES+36 more
Myeloid leukaemia
no clinical trials 3 substances studiedView
- Substances:DaunorubicinPevonedistat+1 more
Myotonic dystrophy
no clinical trials 5 substances studiedView
- Substances:Vx-67016-Base Single Stranded Rna Targeting Mir-23B Linked To Oleic Acid+3 more
Myxofibrosarcoma
no clinical trials 2 substances studiedView
- Substances:TrabectedinTtf-Ngr
Myxoid liposarcoma
no clinical trials 8 substances studiedView
About rare diseases in clinical trials
A disease is called rare in the EU when it affects fewer than 5 in 10 000 people. Individually uncommon, together they affect millions – and most still have no approved treatment.
Because the number of patients is small, trials in rare diseases often recruit across several countries at once. That is why a trial listed here may be worth looking at even when it runs nowhere near you.