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Gene Therapy Candidate Gets Priority Review for Metachromatic Leukodystrophy

September 18, 2023

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

Bobby Gaspar Drug Administration Priority Review Biologics License Application Gross Motor Function Prescription Drug User Fee Act

Source: empr.com

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

May 22, 2023

- 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 States Genevieve Laforet Florian Eichler Bio Pharma Inc Bridgebio Pharma Inc Drug Administration

Source: marketscreener.com

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

April 11, 2022

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

Soult Ukpyolsi South Korea Hyun Jung Chang Eun Sol Jo Hye Kim Sungkyunkwan University

Source: horsetalk.co.nz

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

October 12, 2021

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

Hyun Jung Chang Hye Kim Samsung Medical Center Department Of Physical Medicine Sungkyunkwan University School Of Medicine Department Of Otorhinolaryngology

Source: horsetalk.co.nz

Khondrion announces first patients dosed in 6-month paediatric Phase II study of sonlicromanol for mitochondrial diseases

April 21, 2021

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 patie...

David Daley Jan Smeitink Melissa Gardiner Mary Jane Elliott Radboud Centre For Mitochondrial Medicine Consilium Strategic Communications

Source: globenewswire.com

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Khondrion announces first patients dosed in 6-month paediatric Phase II study of sonlicromanol ...

April 21, 2021

Press release content from Globe Newswire. The AP news staff was not involved in its creation. Khondrion announces first patients dosed in 6-month paediatric Phase II study of sonlicromanol ... KhondrionApril 21, 2021 GMT 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 tha...