New orphans lagging for Aust

Posted 27 May 2022 AM

Four new medicines for rare diseases recommended by Europe's advisory committee, the CHMP, are yet to make a local regulatory appearance, suggesting Australia may be far back on the waitlist.

Among the list is PTC Therapeutics gene therapy Upstaza for AADC deficiency, a fatal rare disorder, with children afflicted by the condition suffering physical, mental and behavioural symptoms, including seizures.

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