Cure Rare Disease Awarded $5.69 Million CIRM Grant to Advance Gene Therapy for Spinocerebellar Ataxia Type 3
Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative ...
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CIRM grant supports Cure Rare Disease’s antisense oligonucleotide therapy for SCA3
Cure Rare Disease has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for ...
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Clinical trial suggests new drug candidate for spinocerebellar ataxia may offer certain therapeutic benefits
However, due to the lack of statistical significance, larger and more robust Phase III trials are necessary ... L-arginine in patients with spinocerebellar ataxia type 6: a multicentre, randomised ...
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SRPT Inks $11B Licensing Deal With Arrowhead for Rare Gene Therapies
These programs target spinocerebellar ataxia type 1, spinocerebellar ataxia type 3 and Huntington's disease. As part of this deal, the companies have also formed a discovery collaboration under ...
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Troriluzole Priority Review Granted for Spinocerebellar Ataxia
The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for troriluzole for the treatment of adults with ...
Biohaven’s troriluzole NDA in spinocerebellar ataxia accepted by the FDA
Biohaven (BHVN) announced that the FDA has accepted for review the company’s New Drug Application, or NDA, for troriluzole for the treatment of ...
Biohaven Gets FDA Priority Review of Troriluzole for Spinocerebellar Ataxia
Biohaven has won Food and Drug Administration priority review for its application seeking approval of troriluzole for adults with spinocerebellar ataxia, a group of rare, genetic neurological ...
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Cure Rare Disease Awarded $5.69 Million CIRM Grant to Advance Gene Therapy for Spinocerebellar Ataxia Type 3
Spinocerebellar ataxia type 3 (SCA3) is a rare neuromuscular disease with a prevalence of one to five in 100,000 people. It is the most common form of spinocerebellar ataxia. The disease is caused ...
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Cure Rare Disease Awarded $5.69 Million CIRM Grant to Advance Gene Therapy for Spinocerebellar Ataxia Type 3
to advance the development of an antisense oligonucleotide therapy for spinocerebellar ataxia type 3 (SCA3), a neurodegenerative disorder with no current treatment.