Mariella Bodemeier Loayza Careaga, PhD | Dec 4, 2023 | 6 min read
Haydeh Payami helped uncover the genetic basis of Parkinson’s disease. Now, she hopes to find new ways to treat the disease by studying the gut microbiome.
The discovery of gene variants in cases of hereditary spastic dysplasia could provide a diagnosis to affected families where no genetic cause could be found before.
Scientists discuss their latest findings on immune cell dynamics, neurodegenerative disease risk factors, and rare cell types obtained from bulk and single cell RNA sequencing experiments.
Victoria Jackson and Michael Yeaman | Apr 30, 2018 | 3 min read
In chapter 4, “Building a Cure Machine,” author Victoria Jackson reveals the challenges in launching a foundation focused on funding research on a rare disease.
Danielle Gerhard, PhD | Mar 22, 2024 | 10+ min read
With the help of directed evolution, scientists inch closer to developing viral vectors that can cross the human blood-brain barrier to deliver gene therapy.
Michael Yeaman and Victoria Jackson | May 1, 2018 | 3 min read
Rarity is a strength, not a weakness, when lessons learned from rare disease patients buoy research and development to find cures for more common diseases.