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Tag: rare disease

First Opinion

Opinion: Take a stand for those who can’t with the Promising Pathway Act

19.05.2022

Every five years since 1992, Congress has reauthorized the Food and Drug Administration user fee package to allow the agency to collect funds from companies that produce human drugs and …

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First Opinion

Opinion: It’s time for Congress to stop blocking mitochondrial replacement therapy

22.04.2022

Australia has become the second country in the world to explicitly authorize and regulate mitochondrial replacement therapies, offering hope to some families who want to have children but fear passing …

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First Opinion

Opinion: Will the shadow of Aduhelm cloud FDA’s decision-making on an ALS therapy?

21.04.2022

On March 30, an FDA advisory panel voted 6 to 4 against recommending approval of what might be the most effective treatment to date for amyotrophic lateral sclerosis. The panel’s …

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Biotech

Virtual Event: CRISPR Quest

24.02.2022

Editor’s note: A livestream of the event is embedded below Last summer Intellia reported the first data showing that CRISPR could be used in the body to combat a disease …

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In the Lab

This family carried a rare mutation that should have been lethal. What was keeping them alive?

15.02.2022

The result didn’t make sense. The researcher kept scanning the mouse’s pregnant belly, back and forth, back and forth. They could see the embryos on the ultrasound screen, cocoon-like, in …

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Health

A mother, shaped by tragedy, embarks on a mission to advance custom medicines

09.02.2022

For weeks, Julia Vitarello avoided the room in her home. The fairy curtains she sewed. The sheets. The quiet. But one day, she placed her desk by the window. There, …

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In the Lab

Provocative new findings suggest a surprising cause of Down syndrome: cells linked to aging

06.01.2022

Down syndrome is the most common genetic disorder, impacting about 1 in 700 newborns around the world. At some point during their first hours and days of embryonic development, their …

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Health / In the Lab

Last-gasp gene therapy saved a Syrian refugee’s life. Five years later, the boy is thriving and a clinical trial is starting

09.12.2021

In the summer of 2015, a 7-year-old named Hassan was admitted to the burn unit of the Ruhr University Children’s Hospital in Bochum, Germany, with red, oozing wounds from head …

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Health

Sequencing whole genomes helps diagnose far more rare diseases, study shows

11.11.2021

When the U.K.’s National Health Service started to use whole genome sequencing, doctors were able to determine diagnoses for more people with rare diseases — including some for whom other …

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