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 ...

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 ...

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 ...

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.

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 ...

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.

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 ...

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