InternationalResearch and clinical trials
Trial of oral branaplam in Huntington's disease stopped early because of nerve side effects
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
Results of a phase 2 study of branaplam, taken by mouth, in Huntington's disease have been published. The drug aimed to reduce production of the faulty protein that causes the disease. In the first group, 18 of the 21 people who took the drug showed signs of damage to the nerves in the hands and feet. For this reason, the study was stopped early. The drug lowered the level of the faulty protein in the spinal fluid.
What does it mean for patients and families?
This drug is not approved for Huntington's disease. The study shows that nerve side effects need to be monitored closely with this type of drug.
Source
Scientific paper: Oral splicing modulator branaplam in Huntington’s disease: a phase 2 randomized controlled trial. Nature Medicine, 2026. DOI: 10.1038/s41591-025-04117-4 · PubMed 41491108
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Huntington disease
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.