Articles with the topic: drug-development

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

Huntington's Disease Therapeutics Conference 2024 - Day 1

Huntington's Disease Therapeutics Conference 2024 - Day 1

Check out research updates from Day 1 of the 2024 HD Therapeutics Conference #HDTC2024

Dr Leora Fox, Dr Rachel Harding, and Dr Sarah HernandezMarch 05, 2024

CRISPR-based drugs: one giant leap for mankind

CRISPR-based drugs: one giant leap for mankind

Casgevy is the first CRISPR-based drug to make its way through the approval process, all but curing Sickle Cell Disease and it’s paving the way for similar drugs targeting other diseases. Is Huntington’s disease next?

Dr Sarah HernandezFebruary 14, 2024

Putting it in print: GENERATION HD1 study results published

Putting it in print: GENERATION HD1 study results published

Data from GENERATION HD1, the Phase 3 clinical trial testing the huntingtin-lowering drug tominersen, have just been published in a scientific journal. The trial ended a while back, so why is this an important milestone, and what’s next?

Dr Leora Fox, Dr Rachel Harding, and Dr Sarah HernandezDecember 07, 2023

Beyond huntingtin lowering: out-of-the-box approaches for the treatment of HD

Beyond huntingtin lowering: out-of-the-box approaches for the treatment of HD

The HD pipeline is rich and varied. Let's talk about some out-of-the-box approaches for developing drugs for HD that don't involve huntingtin lowering.

Kelly AndrewNovember 24, 2023

Drug to treat movement symptoms of HD approved by FDA

Drug to treat movement symptoms of HD approved by FDA

The FDA has approved valbenazine, also known as INGREZZA, as a treatment for the movement symptoms of Huntington’s disease

Dr Leora Fox and Dr Rachel HardingAugust 22, 2023