Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging ...
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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 ...
Rare disease clinical trials are smaller and often use surrogate endpoints, reducing costs but posing unique operational ...
Researchers led by the University of Vienna and Liège University Hospital Center have identified genetic variants associated ...
Data from up to 42 months of treatment reinforce lucerastat’s potential as a first-in-class oral substrate reduction therapy addressing key unmet needs across the Fabry populationThe company is ...
Biotech stocks are in focus as analysts track upcoming clinical data and regulatory decisions in autoimmune and rare disease ...
A Google DeepMind invention that uses artificial intelligence (AI) to predict how DNA mutations behave could have a ...
Activation of immune blood biomarkers from interferon gamma pathway distinguished long-term survivors (>=2 years) ...
Moderna will continue to lead clinical development and manufacturing for mRNA-3927Moderna to receive up to $160 million ...
In todays pharmaceutical executive daily, Mirum Pharmaceuticals strengthens its rare disease portfolio through a $268 million ...
Belite Bio (BLTE) stock outlook: tinlarebant clinical data, 2026 filings, key FDA/EU risks, and $165/share valuation. See if ...
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Genetic variants associated with rare inherited growth disorder identified in two prehistoric individuals
Researchers led by the University of Vienna and Liège University Hospital Centre have identified genetic variants associated with a rare inherited growth disorder in two prehistoric individuals who ...
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