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Results For "Rare-Pediatric-Disease-Designation"

5 News Found

Praxis Precision hits milestone in fight against rare paediatric seizures
Clinical Trials | December 08, 2025

Praxis Precision hits milestone in fight against rare paediatric seizures

The EMBOLD study evaluated relutrigine for patients with SCN2A and SCN8A developmental and epileptic encephalopathies


Lily announces positive gene therapy result for genetic hearing loss
Clinical Trials | January 25, 2024

Lily announces positive gene therapy result for genetic hearing loss

Hearing restoration was observed within 30 days of a single administration of AK-OTOF in the initial AK-OTOF-101 study participant


EMA grants ODD to GC Biopharma's treatment for Sanfilippo Syndrome
Drug Approval | January 23, 2024

EMA grants ODD to GC Biopharma's treatment for Sanfilippo Syndrome

GC1130A has previously achieved notable milestones by securing both RPDD and ODD from the U.S. FDA in January 2023


Wacker Biotech partners with Caeregen Therapeutics on regenerative treatment for vision loss
News | June 28, 2023

Wacker Biotech partners with Caeregen Therapeutics on regenerative treatment for vision loss

As Caeregen’s CDMO partner for CTR-107, Wacker Biotech will produce drug substance at its site in Halle, Germany, and complete clinical trial drug product production at its site in Amsterdam, the Netherlands


Pfizer acquires Global Blood Therapeutics
News | October 09, 2022

Pfizer acquires Global Blood Therapeutics

Acquisition brings leading sickle cell disease portfolio and pipeline to Pfizer with potential to address critical needs in an underserved patient community