PTC518 is an oral medication that reduces the production of the mutated Huntingtin protein that causes disease progression. Programs with Fast Track designation can benefit from early interactions with the FDA, and may be
Prilenia, a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, has filed a European Marketing
I belong to the third generation of a family affected by Huntington’s Disease, but it wasn’t until 2024, thanks to the tireless efforts of my second father, F. Cook, that
Abu Dhabi, UAE; June 19, 2024: IROS, an Abu Dhabi-based contract research organization (part of the M42 group), has partnered with Prilenia Therapeutics, a clinical-stage biotech company, and the International
“We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of NfL in the CSF at 24 months,”
Today, Wave Life Sciences announced positive results from SELECT-HD, our Phase 1b/2a placebo-controlled trial evaluating the investigational therapy WVE-003. These results demonstrate that WVE-003 selectively lowers toxic, mutant huntingtin (mHTT)
At Month 12, mutant Huntingtin (mHTT) in the blood was lowered by 22% and 43% respectively for 5mg and 10mg doses. A similar result was seen in cerebrospinal fluid, where
Earlier this week Sage Therapeutics announced the results of their SURVEYOR study which quantified the cognitive impact of Huntington’s Disease. Using the HD-Cognitive Assessment Battery (HD-CAB) they measured the difference
IHA President Svein Olaf Olsen was keynote speaker on Huntington’s Disease at the MENA Congress for Rare Diseases in Abu Dhabi last month. He spoke of the urgent need to
Today we are very happy in the Huntington community. The announcement from Uniqure that the company has received the Regenerative Medicine Advanced Therapy (RMAT) “Designation for Investigational Gene Therapy AMT-130
PTC518 is an oral medication that reduces the production of the mutated Huntingtin protein that causes disease progression. Programs with Fast Track designation can benefit from early interactions with the FDA, and may be
Prilenia, a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, has filed a European Marketing
I belong to the third generation of a family affected by Huntington’s Disease, but it wasn’t until 2024, thanks to the tireless efforts of my second father, F. Cook, that
Abu Dhabi, UAE; June 19, 2024: IROS, an Abu Dhabi-based contract research organization (part of the M42 group), has partnered with Prilenia Therapeutics, a clinical-stage biotech company, and the International
“We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of NfL in the CSF at 24 months,”
Today, Wave Life Sciences announced positive results from SELECT-HD, our Phase 1b/2a placebo-controlled trial evaluating the investigational therapy WVE-003. These results demonstrate that WVE-003 selectively lowers toxic, mutant huntingtin (mHTT)
At Month 12, mutant Huntingtin (mHTT) in the blood was lowered by 22% and 43% respectively for 5mg and 10mg doses. A similar result was seen in cerebrospinal fluid, where
Earlier this week Sage Therapeutics announced the results of their SURVEYOR study which quantified the cognitive impact of Huntington’s Disease. Using the HD-Cognitive Assessment Battery (HD-CAB) they measured the difference
IHA President Svein Olaf Olsen was keynote speaker on Huntington’s Disease at the MENA Congress for Rare Diseases in Abu Dhabi last month. He spoke of the urgent need to
Today we are very happy in the Huntington community. The announcement from Uniqure that the company has received the Regenerative Medicine Advanced Therapy (RMAT) “Designation for Investigational Gene Therapy AMT-130
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