News

Breakthrough represents a new hope for HD patients

Scientists from the Weizmann Institute in Israel have published the results of an animal study which holds tremendous promise for the treatment of Huntington’s Disease.  They have discovered that two small molecules, called SPI-24 and SPI-77, can effectively reduce mutant huntingtin expression in the brain while leaving the normal gene

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uniQure Announces Update on Phase I/II Clinical Trials of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

~ Patients treated with AMT-130 continue to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to an inclusion criteria-matched natural history of the disease ~ ~ Mean CSF NfL continue to demonstrate favorable trends with low-dose patients below baseline at 30 months and high-dose patients near baseline

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Breakthrough represents a new hope for HD patients

Scientists from the Weizmann Institute in Israel have published the results of an animal study which holds tremendous promise for the treatment of Huntington’s Disease.  They have discovered that two small molecules, called SPI-24 and SPI-77, can effectively reduce mutant huntingtin expression in the brain while leaving the normal gene

Read More »

uniQure Announces Update on Phase I/II Clinical Trials of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

~ Patients treated with AMT-130 continue to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to an inclusion criteria-matched natural history of the disease ~ ~ Mean CSF NfL continue to demonstrate favorable trends with low-dose patients below baseline at 30 months and high-dose patients near baseline

Read More »
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