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Hunter syndrome

PharmaDeviceNews Hunter syndrome
JCR Pharmaceuticals’ IZCARGO UAE marketing authorization expands access to a blood-brain barrier-penetrating enzyme replacement therapy for Hunter syndrome. Representative image.
Pharma & Biotech

IZCARGO has moved beyond Japan, but JCR Pharmaceuticals’ real test begins after the UAE authorization

JCR Pharmaceuticals’ UAE authorization for IZCARGO opens a new market, but reimbursement, supply and Phase III evidence will shape its impact.

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A scientist reviews brain imaging and neurological research data as Denali Therapeutics channels its $195 million FDA voucher proceeds into blood-brain barrier drug development and the TransportVehicle pipeline. Representative image.
Pharma & Biotech

Denali monetises its FDA voucher as AVLAYAH enters a critical confirmatory phase

Denali Therapeutics is converting AVLAYAH’s regulatory reward into non-dilutive capital for its blood-brain barrier pipeline, but clinical and commercial risks remain.

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Pharma & Biotech

Is JCR unlocking the CNS for lysosomal storage diseases before gene therapy gets there?

Find out how JCR Pharmaceuticals is redefining lysosomal disease therapy with brain-targeted delivery platforms.

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