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Gene Therapy Candidate Gets Priority Review for Metachromatic Leukodystrophy

The FDA has accepted for Priority Review the BLA for OTL-200 for the treatment of metachromatic leukodystrophy.

Bobby gasparDrug administrationPriority reviewBiologics license applicationGross motor functionPrescription drug user fee act

Orchard Therapeutics Announces Presentation of Data Comprising the Clinical Package for the OTL-200 BLA in MLD at the SSIEM Annual Symposium 2023

Orchard Therapeutics Announces Presentation of Data Comprising the Clinical Package for the OTL-200 BLA in MLD at the SSIEM Annual Symposium 2023
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Israel generalUnited kingdomCity ofUnited statesBenjamin navonLeslie meltzerExchange commissionSan raffaele telethon institute for gene therapyDrug administrationEuropean commissionEuropean unionTelethon institute for gene therapyTherapeutics announces presentationData comprisingClinical packageInborn errors

BridgeBio Pharma Presents Updated Encouraging Clinical and Biomarker Data from its BBP-812 Canavan Disease Gene Therapy Program at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

- Following treatment, the N-acetylaspartate levels of CANaspire participants are consistent with levels seen in individuals with milder Canavan disease based on findings from the Company’s. | May 22, 2023

United statesGenevieve laforetFlorian eichlerBio pharma incBridgebio pharma incDrug administrationEuropean unionExchange commissionEuropean medicines agencySafety monitoring committeeSecurities exchangeGross motor functionBio pharmaAspa therapeuticsMassachusetts general hospitalRare neurological disease

Children with cerebral palsy benefited from a riding simulator coupled with virtual reality

Study in South Korea reported improvements in overall motor function and increases in skeletal muscle mass.

Soult ukpyolsiSouth koreaHyun jung changEun sol joHye kimSungkyunkwan universityHorse riding simulatorVirtual realityGross motor functionBody compositionCerebral palsyCreative commons license

Researchers add new twist to a riding simulator for children with cerebral palsy

Youngsters wore a virtual reality headset while using a horse-riding simulator.

Hyun jung changHye kimSamsung medical centerDepartment of physical medicineSungkyunkwan university school of medicineDepartment of otorhinolaryngologyIncorporated horse riding simulator to improve motorJung changSungkyunkwan university schoolPediatric balance scaleGross motor functionYoung sook parkChang woo kimPhysical medicineSamsung changwon hospitalYong gi jung

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