Articles with the topic: drug-development

Moving into the Fast Lane: uniQure and the FDA Are on the Same Track for Accelerated Approval

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.

Dr Rachel Harding and Dr Sarah HernandezDecember 10, 2024

Bringing HD Treatments to Market: The Role of Regulatory Oversight

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.

Dr Rachel HardingOctober 21, 2024

Highlighting a link between brain disorders on Ataxia Awareness Day

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.

Dr Leora FoxSeptember 25, 2024

Hope vs. hype: seeking truth in recent Prilenia headlines

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?

Dr Sarah HernandezSeptember 10, 2024

Blue skies for Skyhawk: Positive news from Phase 1 trial for SKY-0515

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.

Dr Sarah HernandezJuly 11, 2024

No pivot needed for PTC-518

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.

Dr Sarah HernandezJune 21, 2024

SURVEYOR opens the door for drugs that treat cognition

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.

Dr Leora FoxJune 12, 2024

A sprinkling of good news for the treatment of HD chorea

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.

Dr Leora FoxMay 03, 2024

Huntington's Disease Therapeutics Conference 2024 - Day 2

Huntington's Disease Therapeutics Conference 2024 - Day 2

Dr Leora Fox, Dr Sarah Hernandez, and Dr Rachel HardingMarch 06, 2024

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