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.
Source: empr.com
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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
Source: marketscreener.com
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.
Source: horsetalk.co.nz
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.
Source: horsetalk.co.nz
Khondrion announces first patients dosed in 6-month paediatric Phase II study of sonlicromanol for mitochondrial diseases
Khondrion announces first patients dosed in 6-month paediatric Phase II study of sonlicromanol for mitochondrial diseases April 21, 2021 07:00 ET | Source: Khondrion Khondrion Study will examine of sonlicromanol, candidates in development for mitochondrial disease NIJMEGEN, the Netherlands – 21 April 2021: Khondrion, a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that the first patients have been successfully dosed in its KHENERGYC paediatric Phase II study (NCT04846036) to explore...
Source: globenewswire.com