Articles with the topic: featured

Mini brains grown in a dish shed light on Huntington’s disease and how we might treat it

Mini brains grown in a dish shed light on Huntington’s disease and how we might treat it

Exciting new findings using 3D human lab-grown mini brains inform ongoing HTT-lowering trials and suggest that stem cell transplants for HD may improve cell-to-cell communication and reduce disease features.

Dr Sarah HernandezAugust 23, 2024

Hereditary Disease Foundation (HDF) conference 2024 – Day 4

Hereditary Disease Foundation (HDF) conference 2024 – Day 4

Read our live tweet compilation from clinical trials and scientific research on Huntington’s disease from Day 4 of the 2024 HDF Milton Wexler Biennial Symposium #HD2024

Dr Sarah Hernandez and Dr Rachel HardingAugust 13, 2024

Hereditary Disease Foundation (HDF) conference 2024 – Day 3

Hereditary Disease Foundation (HDF) conference 2024 – Day 3

Read our live tweet compilation from clinical trials and scientific research on Huntington’s disease from Day 3 of the 2024 HDF Milton Wexler Biennial Symposium #HDF2024

Dr Rachel Harding and Dr Sarah HernandezAugust 12, 2024

Hereditary Disease Foundation (HDF) conference 2024 – Day 2

Hereditary Disease Foundation (HDF) conference 2024 – Day 2

Read updates from clinical trials and scientific research on Huntington’s disease from Day 2 of the 2024 HDF Milton Wexler Biennial Symposium #HDF2024

Dr Tamara Maiuri, Dr Sarah Hernandez, and Dr Rachel HardingAugust 10, 2024

Hereditary Disease Foundation (HDF) conference 2024 – Day 1

Hereditary Disease Foundation (HDF) conference 2024 – Day 1

Read updates from clinical trials and scientific research on Huntington’s disease from Day 1 of the 2024 HDF Milton Wexler Biennial Symposium #HDF2024

Dr Rachel Harding, Dr Tamara Maiuri, and Dr Sarah HernandezAugust 09, 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

Positive news from Wave Life Sciences SELECT-HD trial

Positive news from Wave Life Sciences SELECT-HD trial

The outcomes of the SELECT-HD clinical trial are out, with encouraging news for this allele-selective huntingtin lowering therapy

Dr Rachel HardingJune 28, 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

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