A spoonful of branaplam helps the huntingtin go down
Branaplam was originally designed to treat spinal muscular atrophy, but a new paper outlines how it could hold promise for treating Huntington’s. This oral drug lowers huntingtin protein and will now be tested in a study called VIBRANT-HD.
HDBuzz have released an article about Novartis' developments with the drug branaplam. After completing their phase I study which was to initially test safe dosing in adults, we find out what is in the pipeline for the phase IIb study, VIBRANT-HD.
This trial will be the first time it is given to adults with Huntington's. Unlike other huntingtin-lowering drugs that are given via injection or spinal tap, branaplam will be administrated as an oral liquid which is much less invasive.
"The team measured huntingtin protein levels when different amounts of branaplam was dosed in cells in a dish and showed that the more drug was given, the more the level of huntingtin protein was lowered." - HDBuzz
Branaplam was originally developed for a fatal childhood disorder called spinal muscular atrophy (SMA) because it changes the levels of a protein called SMN2, which underlies that disease.
Branaplam was originally designed to treat spinal muscular atrophy, but a new paper outlines how it could hold promise for treating Huntington’s. This oral drug lowers huntingtin protein and will now be tested in a study called VIBRANT-HD.
HDBuzz have released an article about Novartis' developments with the drug branaplam. After completing their phase I study which was to initially test safe dosing in adults, we find out what is in the pipeline for the phase IIb study, VIBRANT-HD.
This trial will be the first time it is given to adults with Huntington's. Unlike other huntingtin-lowering drugs that are given via injection or spinal tap, branaplam will be administrated as an oral liquid which is much less invasive.
"The team measured huntingtin protein levels when different amounts of branaplam was dosed in cells in a dish and showed that the more drug was given, the more the level of huntingtin protein was lowered." - HDBuzz
Branaplam was originally developed for a fatal childhood disorder called spinal muscular atrophy (SMA) because it changes the levels of a protein called SMN2, which underlies that disease.