Clinical trial provides hope for Mass. child with rare genetic disorder
A RARE AND FATAL DEGENERATIVE DISEASE THAT CAUSES PROGRESSIVE DAMAGE TO NERVE CELLS AND LOSS OF WHITE MATTER IN THE BRAIN. GETTING THAT DIAGNOSIS WHEN FAMILIES WOULD GET THAT, THE DOCTORS WOULD GIVE ...
Discovery at Massachusetts General Hospital creates roadmap for a rare disease
Rithvik wasn’t dealt an easy hand in life. His first symptom — a distended abdomen — was identified during his birth ...
Correcting Genetic Spelling Errors With Next-Generation Crispr
Treatments for rare diseases are hard to create and expensive to deliver, but there is new hope for editing the software of ...
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Rare Disease R&D: one step forward, but a long road ahead
As a result, even the most basic tasks, such as correctly identifying such a disease, can be made extremely ... genetic root causes addressed.” Being able to ascertain the genetic root of a rare ...
Healthy Returns: The FDA approved fewer new drugs in 2024 — but some big ones hit the market
The FDA wrapped up 2024 with 50 new drug approvals, clearing a wave of long-awaited treatments. Meanwhile, Transcarent will take Accolade private.
A Virtual Cell Is a ‘Holy Grail’ of Science. It’s Getting Closer.
Large language models may unlock a new and valuable type of research.
5 things we know and still don't know about COVID, 5 years after it appeared
It can take several weeks to bounce back after a bout of COVID-19, but some people develop more persistent problems. The ...
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Biotech Stocks Prepare For Action In 2025. Weight-Loss Drugs, AI And Trump 2.0 Are The Catalysts.
Biotech stocks are poised for an exciting 2025 as experts watch for new weight-loss drugs, AI's impact and Trump's second ...