✉news WorldEU Politics first seen 14 h ago, last 4 h ago, peak #9
EU approves first drug for cerebral adrenoleukodystrophy
Original: European Commission grants marketing authorization for NEZGLYAL® (leriglitazone), the first pharmacological treatment approved for cerebral adrenoleukodystrophy (cALD), a rare neurodegenerative disease
The European Commission has granted marketing authorization for NEZGLYAL (leriglitazone), making it the first pharmacological treatment approved for cerebral adrenoleukodystrophy, a rare and rapidly progressive neurodegenerative disease that primarily affects young boys. Until now, care options for cALD were limited mainly to stem cell transplantation in eligible patients. The approval is being highlighted as a significant milestone for families affected by the rare disease and for rare-disease drug development in Europe.
Why now: The approval offers the first-ever approved medicine for a devastating rare disease with previously no pharmacological treatment.
European CommissionNEZGLYALleriglitazonecerebral adrenoleukodystrophy
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