Rare & Genetic Diseases
| 4 min read
The therapy lengthened telomeres and improved immune function in people with telomere biology disorder, showing promise for treating bone marrow failure safely.
| 3 min read
A new compound, Vykat XR, addresses insatiable hunger in the rare genetic condition, offering relief for patients and hope for families.
| 4 min read
In a Phase 2b trial, Khondrion’s sonlicromanol led to significant health benefits for patients with mitochondrial disease linked to a common mutation. 
| 3 min read
A gene therapy targeting organs beyond the brain could improve quality of life and possibly extend survival in children with the neurodegenerative disease.
| 3 min read
An enzyme reconstructed from a three-billion-year-old bacterium widens gene editing capabilities to even more regions of the genome.
| 5 min read
Michelle Werner and her team at Alltrna are developing novel tRNA therapeutics to overwrite mutations shared by hundreds of rare genetic diseases.
| 4 min read
People and mice with ALS-linked mutations show altered sleep patterns early in life. Modifying sleep hormone levels normalized sleep cycles in mice.
| 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.
Discover new advancements in modeling amyotrophic lateral sclerosis.
| 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.
| 7 min read
Achieving higher accuracy and depth in bottom-up proteomics involves navigating a multifaceted process with various steps and challenges.
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