Genetic Diseases Inborn
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-08-26EMA Authorised: Vijoice (alpelisib) — Novartis Europharm Limited
EMA authorised Vijoice (alpelisib) by Novartis Europharm Limited with conditional approval for genetic diseases of growth disorders; orphan …
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EMA approval · 2026-08-13EMA Authorised: Pyrukynd (mitapivat sulfate) — Agios Netherlands B.V.
Pyrukynd (mitapivat sulfate) has been authorised in the EU for genetic diseases inborn and hemolytic anemia, with orphan medicine designatio…
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EMA approval · 2026-07-09EMA Authorised: Pyrukynd (mitapivat sulfate) — Agios Netherlands B.V.
Pyrukynd (mitapivat sulfate) has been authorised in the EU for genetic diseases inborn and hemolytic anemia, orphan medicine designation.
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CLINICAL_TRIALS research · 2025-03-06Clinical Trial Update: Evaluating Prenatal Exome Sequencing Study
A prenatal exome sequencing study conducted by Leiden University Medical Center is currently active but not recruiting, investigating geneti…
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EMA withdrawal · 2024-03-14EMA Application withdrawn: Vijoice (alpelisib) — Novartis Europharm Limited
The EMA has withdrawn the application for Vijoice (alpelisib) from Novartis Europharm Limited for genetic diseases and growth disorders.
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