Summary
- Update from Prilenia: Pursuing pridopidine for HD
- The Buzz on brains: What does HD do on a cellular level?
- Participate in an Interview Study: Patient Supported Approaches to Gene Editing (PaSAGE)
Update from Prilenia: Pursuing pridopidine for HD
Earlier this week, Prilenia issued a press release and community letter announcing their intent to submit a Marketing Authorization Application (MAA) in the EU for Pridopidine in HD.
This application will be submitted to the European Medicines Agency, the equivalent of the FDA in Europe. It does not mean that the drug will be approved; it means that the EMA will evaluate the science and the data to make a decision. Prilenia also notes that they will have upcoming discussions with the FDA about a path forward in the USA.
Clinical trials of pridopidine for people with HD have not met their key goals. However, the drug is safe, and the data from some pre-specified analyses in the study might suggest some benefit in the first year for certain participants. This is what has led Prilenia to pursue approval. They shared a comprehensive list of frequently asked questions as part of their community letter, which you can read here.
The Buzz on brains: What does HD do on a cellular level?
Certain parts of the brain are more prone to damage from HD than others, but there is still much to learn as to why. Researchers at Rockefeller University have been studying this phenomenon in individual cells from different regions of human brains generously donated by people with and without HD. In their most recent article, the HDBuzz team took a deep-dive into the group’s recently published findings—you can read their take in full here. It is important to note that critical studies such as these are only made possible by the generous contributions made by people with HD to support HD science: precious post-mortem brain samples. If you’re interested in learning about how this amazing gift can improve our understanding of HD or how to participate, click here or visit HDTrialfinder.org.
Participate in an Interview Study: Patient Supported Approaches to Gene Editing (PaSAGE)
The Patient Supported Approaches to Gene Editing (PaSAGE) Study is an NIH-funded study about views, ethical concerns, and clinical aspirations on prenatal gene editing technologies. This is a group of social scientists and bioethicists studying the possible impact on families and communities of genetic therapies that might become available in the future. They are interested in connecting with people from HD families in the USA who have lived experience with HD, whether as a patient, caregiver or loved one.
Participation in this interview study involves a one-hour phone call with someone from the research team at the Mayo clinic. If you have a family history of HD, they would like to know your thoughts. No matter how you feel about potential treatments, they are interested in hearing from you. If you would like to take part in this interview study, please email pasagestudy@mayo.edu.
