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15 Products
118 Diseases
15 Products
43 Trials
3365 News
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Active industry trials
Active owner-sponsored trials
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Translarna
(ataluren) / PTC Therap
Evrysdi
(risdiplam) / SMA Foundation, PTC Therap, Roche
Vincerinone
(vatiquinone) / Sumitomo Pharma, PTC Therap
Tegsedi
(inotersen) / Ionis, SOBI
Waylivra
(volanesorsen) / Novartis, SOBI
Emflaza
(deflazacort) / Marathon, PTC Therap
synthetic sepiapterin
(PTC923 PKU) / PTC Therap
emvododstat
(PTC299) / PTC Therap
unesbulin
(BMIi-1) / PTC Therap
Upstaza
(eladocagene exuparvovec) / PTC Therap
PTC518
/ PTC Therap
R-troloxamide quinone
(EPI-589) / Sumitomo Pharma
utreloxastat
(PTC857) / PTC Therap
RG7800
/ Roche
