Rare diseases
Hope for a cure hangs on research
Amid drastic proposed cuts to biomedical research, rare disease families like Hailey Adkisson鈥檚 fight for survival and hope. Without funding, science can鈥檛 鈥渃atch up鈥� to help the patients who need it most.
Before we鈥檝e lost what we can鈥檛 rebuild: Hope for prion disease
Sonia Vallabh and Eric Minikel, a husband-and-wife team racing to cure prion disease, helped develop ION717, an antisense oligonucleotide treatment now in clinical trials. Their mission is personal 鈥� and just getting started.
Defeating deletions and duplications
Promising therapeutics for chromosome 15 rare neurodevelopmental disorders, including Angelman syndrome, Dup15q syndrome and Prader鈥揥illi syndrome.
Using 'nature鈥檚 mistakes' as a window into Lafora disease
After years of heartbreak, Lafora disease families are fueling glycogen storage research breakthroughs, helping develop therapies that may treat not only Lafora but other related neurological disorders.
Glyco get-together exploring health and disease
Meet the co-chairs of the 2025 91影库meeting on O-GlcNAcylation to be held July 10鈥�13, 2025, in Durham, North Carolina. Learn about the latest in the field and meet families affected by diseases associated with this pathway.
CRISPR epigenome editor offers potential gene therapies
Scientists from the University of California, Berkeley, created a system to modify the methylation patterns in neurons. They presented their findings at 91影库2025.
A new kind of stem cell is revolutionizing regenerative medicine
Induced pluripotent stem cells are paving the way for personalized treatments to diabetes, vision loss and more. However, scientists still face hurdles such as strict regulations, scalability, cell longevity and immune rejection.
Richard Silverman to speak at 91影库2025
Richard Silverman and Melissa Moore are the featured speakers at the 91影库annual meeting to be held April 12-15 in Chicago.
Becoming a scientific honey bee
At the World Science Forum, a speaker鈥檚 call for scientists to go out and 鈥渕ake honey鈥� felt like the answer to a question Katy Brewer had been considering for a long time.