PROM-Ataxia published (CCAS and PROM being used in natural history studies and clinical trials of ataxia)
The Schmahmann Lab together with the National Ataxia Foundation and with support from Biohaven Pharma developed the Patient Reported Outcome Measure for Ataxia (PROM-Ataxia). This is the first patient-derived reporting checklist of symptoms and challenges faced by patients with cerebellar ataxia, which provides critical and previously unavailable insights into the ataxias and their course over time. The PROM-Ataxia has been incorporated into natural history studies and clinical trials in ataxia, and is being translated into multiple languages for use internationally.
Enrollment for the Troriluzole (SCA) and Verdiperstat (MSA) studies has closed and are expected to be completed by the summer of 2022
Enrollment has now closed in Biohaven’s troriluzole study in people with spinocerebellar ataxia, and the Biohaven verdiperstat study in those with multiple system atrophy. The MGH Ataxia Center is a lead site internationally in both these studies, and as we draw nearer to the completion of these studies we remain hopeful that the new medications will produce symptomatic benefit and even some slowing of disease.
Biogen tapped MGH Ataxia Center to be a lead clinical trial suite for the new ASO in SCA3
Hot off the press news is that Biogen has tapped the MGH Ataxia Center to be a lead clinical trial suite for the new gene-related treatment of spinocerebellar ataxia type 3, Machado Joseph Disease, using antisense oligonucleotides (ASOs). SCA3 is the commonest ataxia worldwide, and it takes a heavy toll on our patient population because it affects so many families in New England. ASOs interrupt the translation of genetic information from DNA through RNA and into the bad proteins that cause the damage in SCA3 and other ataxic disorders. This is an exciting new horizon in the treatment of genetic disorders, and we are very excited to participate in the clinical trials of this novel therapy in ataxia patients and their families. SCA3 now, other ataxias next…
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