Compound Family

Rare Disease dossiers

Orphan drug therapeutics addressing genetic and ultra-rare conditions with precision mechanisms.

Showing 10 of 10 compounds
Amondys 45
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Rare Disease

Amondys 45

Pharmaceutical medication (Casimersen)

TherapeuticClinical Research
Cablivi
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Rare Disease

Cablivi

Pharmaceutical medication (Caplacizumab-yhdp)

TherapeuticClinical Research
Empaveli
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Rare Disease

Empaveli

Pharmaceutical medication (Pegcetacoplan)

TherapeuticClinical Research
Evrysdi
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Rare Disease

Evrysdi

Pharmaceutical medication (Risdiplam)

TherapeuticClinical Research
Exondys 51
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Rare Disease

Exondys 51

Pharmaceutical medication (Eteplirsen)

TherapeuticClinical Research
Spinraza
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Rare Disease

Spinraza

Pharmaceutical medication (Nusinersen Sodium)

TherapeuticClinical Research
Tavneos
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Rare Disease

Tavneos

Pharmaceutical medication (Avacopan)

TherapeuticClinical Research
Ultomiris
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Rare Disease

Ultomiris

Pharmaceutical medication (Ravulizumab-cwvz)

TherapeuticClinical Research
Viltepso
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Rare Disease

Viltepso

Pharmaceutical medication (Viltolarsen)

TherapeuticClinical Research
Vyondys 53
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Rare Disease

Vyondys 53

Pharmaceutical medication (Golodirsen)

TherapeuticClinical Research