The crisis facing rare disease and disability families in Connecticut is caused by systemic underinvestment, administrative barriers, and policy choices.
DiMe introduces core digital measures set to accelerate drug development for pediatric rare diseases
The Digital Health Measurement Collaborative Community (DATAcc) by the Digital Medicine Society (DiMe) today announced the ...
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CIRM approves new $100 million plan to accelerate genetic therapies for patients with rare diseases
The California Institute for Regenerative Medicine (CIRM) has approved a new funding program to accelerate therapies for rare diseases, which sets aside $100 million over two years to create a ...
A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...
Morning Overview on MSN
Ancient DNA uncovers 12,000-year-old case of rare genetic disorder
A teenager who lived in southern Europe around 12,000 years ago has become the earliest person in history to receive a ...
To be only six years old and have seven breaks and two surgeries, that’s a lot,” Jessica said. “Some days are really hard.” ...
Add Yahoo as a preferred source to see more of our stories on Google. U.S. Secretary of Health and Human Services Robert F. Kennedy Jr. speaks with U.S. Vice President JD Vance (not pictured) during ...
Add Yahoo as a preferred source to see more of our stories on Google. Catherine Illingworth, 39, knew something was wrong the moment her son's neurologist walked into the room. Illingworth requested ...
Rare diseases are genetic disorders that affect a small proportion of the population. Despite their rarity, they can be a ...
Researchers led by the University of Vienna and Liège University Hospital Center have identified genetic variants associated ...
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