GlobalResearch and clinical trials
Add-on sotatercept reduced serious events in high-risk PAH in a phase 3 trial
The trial included 172 PAH patients at high risk of death.
Peer-reviewed publicationPAH
Rare Diseases
News
New treatments, research and health policy from around the world and from Türkiye. Each with its source and level of evidence.
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United KingdomResearch and clinical trials
Data from England: in pyruvate kinase deficiency, the risk of death is five times higher than in peers
In England, 89 patients with pyruvate kinase deficiency were identified from health records.
Peer-reviewed publicationPK deficiency
EuropeResearch and clinical trials
International study of 658 patients with ARPKD: a score predicting kidney risk at two months of age developed
In a study led by Germany, with centers from Türkiye also taking part, up to 658 patients with ARPKD were studied.
Peer-reviewed publicationARPKD
EuropeResearch and clinical trials
Blood pressure drug irbesartan reduced vascular events over two years in vascular EDS
Irbesartan was tested in a trial with 57 adults with vascular EDS.
Peer-reviewed publicationVascular EDS
GlobalResearch and clinical trials
Gene therapy given to one eye in LHON: similar improvement seen in both eyes over 5 years
A gene therapy in patients with LHON due to an MT-ND4 gene change was followed for 5 years.
Peer-reviewed publicationLHON
GlobalResearch and clinical trials
Lerodalcibep phase 3 trial in HoFH: expected equivalence not shown on the main goal
In six countries, including Türkiye, 66 HoFH patients took two drugs one after the other.
Peer-reviewed publicationHoFH