✉news WorldEU Politics first seen 4 h ago, last 1 h ago, peak #11
EU approves NEZGLYAL, first drug for childhood cerebral ALD
Original: European Commission grants marketing authorisation for NEZGLYAL, first pharmacological treatment for cALD
The European Commission has granted marketing authorisation for NEZGLYAL, the first pharmacological treatment for childhood cerebral adrenoleukodystrophy (cALD), a rare and severe genetic brain disorder. Until now, treatment options were largely limited to stem cell transplantation and supportive care. The approval offers a new therapy option for patients across the EU and is being noted in rare disease and biotech circles.
Why now: It is the first-ever approved medicine for a rare, devastating childhood disease, making it major news in rare disease and EU regulatory circles.
European CommissionNEZGLYALchildhood cerebral adrenoleukodystrophyEuropean Union
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Evidence
- European Commission grants marketing authorisation for NEZGLYAL, first pharmacological treatment for cALD · BioSpectrum Asia
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