Bioblast Pharma Ltd. ORPN announced positive results from a phase IIa study on its lead candidate, trehalose, for the treatment of patients with spinocerebellar ataxia type 3 (SCA3). A look at ...
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YOKOHAMA, Japan, June 24, 2026 /PRNewswire/ -- REPROCELL Inc. today announced the submission of an application for manufacturing and marketing approval in Japan for the regenerative medicine product ...
WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the ...
Collaborating with the University of Oxford, Professor Ho Yin Edwin Chan's research team from the School of Life Sciences of The Chinese University of Hong Kong (CUHK) recently unveiled the ...
Niigata, Japan – Using machine learning, the Department of Neurology at Niigata University has developed a model to predict the asymptomatic probability at each age from the current age and number of ...
Vico Therapeutics has started patient dosing in an expanded cohort under a twice-yearly regimen for its Phase I/IIa clinical trial of VO659 in Huntington’s disease (HD), spinocerebellar ataxia type 3 ...
Mayo Clinic researchers, along with national and global collaborators, have developed a potential test for Machado-Joseph disease, or spinocerebellar ataxia type 3 (SCA3)—a disease that has no cure.
Spinocerebellar ataxia type 3/Machado–Joseph disease (SCA3/MJD) is a progressive autosomal dominant neurodegenerative disease caused by abnormal CAG repeats in the exon 10 of ATXN3. The accumulation ...
This pilot study was initiated to show the existence of founder effects in the Dutch autosomal dominant cerebellar ataxia (ADCA) population. The ADCAs comprise a clinically heterogeneous group of ...
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 14, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with ...
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