drug-development
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Full Steam Ahead: uniQure’s On Track With Hope for Accelerated Approval of Huntington’s Disease Drug
uniQure has aligned with the US FDA on next steps to achieve accelerated approval of AMT-130. If data remain positive, they will be on track to market the first HD gene therapy.
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Stopping C-A-G Repeat Expansion In Its Tracks
A new study shows that lowering MSH3, a key DNA repair protein, with antisense oligonucleotide (ASO) therapy can stop CAG repeat expansions in HD patient-derived brain cells. This could be a promising way to slow symptom onset and progression.
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Moving into the Fast Lane: uniQure and the FDA Are on the Same Track for Accelerated Approval
In an update shared today, uniQure announced alignment with the US drug regulator on key criteria for accelerated approval of drugs for Huntington’s disease.
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Bringing HD Treatments to Market: The Role of Regulatory Oversight
Moving drugs from the lab bench to pharmacy shelves is no small task. In this article we delve into the role regulatory oversight plays in clinical trials and the approval of medicines for Huntington’s disease.
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Highlighting a link between brain disorders on Ataxia Awareness Day
September 25th is Ataxia Awareness Day, so HDBuzz is highlighting the connections between HD and Ataxia research. Learn about how these fields have historically grown in parallel and informed one another.
By Dr Leora Fox -
Hope vs. hype: seeking truth in recent Prilenia headlines
Although pridopidine has suffered four negative trials for HD, the message from Prilenia continues to be positive. What is hope and what is hype in this sixteen-year quest for regulatory approval?
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Blue skies for Skyhawk: Positive news from Phase 1 trial for SKY-0515
There’s more good news in the forecast in the Huntington’s disease therapeutic space as we receive positive results from Skyhawk Therapeutics about their small molecule SKY-0515 that lowers huntingtin and targets somatic expansion.
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No pivot needed for PTC-518
PTC Therapeutics shared 12 month data from the PIVOT-HD trial, testing the oral HTT lowering drug PTC-518. While designed to assess safety, they shared encouraging results that the drug showed promising signs for biomarkers and some clinical metrics.
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SURVEYOR opens the door for drugs that treat cognition
Sage Therapeutics announced results from SURVEYOR, a trial that looked at cognitive changes in people with HD, and tested short-term safety of dalzanemdor. The study was small but met key goals, and additional trials are in progress.
By Dr Leora Fox -
A sprinkling of good news for the treatment of HD chorea
A new form of the chorea drug valbenazine (INGREZZA) has been approved by the United States Food and Drug Administration (FDA) for those who have difficulty swallowing pills.
By Dr Leora Fox