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Rare & Genetic Diseases
| 6 min read
Enzyme replacement therapy has been the mainstay of Pompe disease. New and emerging therapies may provide more options for patients.
| 3 min read
New dual AAV vector delivery overcomes challenges in delivering large genes.
| 3 min read
The gene therapy treats the rare genetic disease AADC deficiency by delivering a functional gene into the brain.
Learn how stem cell-derived motor neurons and microglia are opening new pathways to understand ALS and explore potential therapies.
| 3 min read
A small percentage of cells in healthy breast tissue show chromosomal abnormalities often linked to cancer, a finding with potential diagnostic implications.
| 3 min read
The FDA approved two new medications for Niemann-Pick disease type C that improve symptoms, showing the power of community advocacy.
Learn practical strategies for using artificial intelligence to find the best drug candidate.
Advanced gene editing, delivery, and analytical tools are driving better gene therapies.
| 9 min read
Rett syndrome currently has no cure. Now, innovations in gene therapy bring the possibility of treating the root cause of the disease closer than ever before.
Emerging CRISPR screening methods are shaping what’s possible in drug development and precision medicine.
| 9 min read
Drug repurposing trials for ichthyosis reveal complexity in the long-untreated disease.
| 4 min read
Previous genome editing techniques only work when delivered early in life. This approach could help preserve hearing that is lost later on.
