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Tag: rare disease

Health / In the Lab

In a first, children with rare genetic diseases get mitochondrial transplants from their mothers

21.12.2022

At a far distant point in Earth’s ancient past, two separate, single-celled life forms — an archaeon and a bacteria — became one in an act either of symbiosis or …

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Biotech / Health

‘All the tech in the world doesn’t solve this’: Rare disease experts push biopharma on equity

08.12.2022

Over the past few years, the biopharmaceutical industry has revved up efforts to diversify clinical trials. But clinical trials for rare diseases are still too often homogeneous. Rare disease experts …

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Biotech / In the Lab / Pharma

On the long road to treating Huntington’s genetic stutter, scientists return to overlooked clues

30.11.2022

In the spring of 1981, a geneticist from Indianapolis and a neurologist from Chicago got in a car and for three days they crisscrossed the yellowing cornfields of Iowa. Every …

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Pharma

House Democrat presses bill to encourage more diversity in clinical trials run by NIH

19.09.2022

Rep. Robin Kelly admits that the word “diversity” has scared some of her Republican colleagues away from her effort to improve representation in clinical trials. “It’s been a little tough …

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Health

At a fashion show for people with spinal muscular atrophy, models take back their own stories

16.09.2022

NEW YORK — The scene: a fashion show on Fifth Avenue. The catwalk was not elevated, as it usually would be. People were packed tightly on benches alongside it in …

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First Opinion

Opinion: The FDA needs to be more flexible in assessing treatments for rare diseases, like the one that seemed to help my son

07.09.2022

Every time I read about clinical trials testing possible treatments for rare diseases, I think of my son, Ty, whose brief but successful foray into such a trial highlights their …

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First Opinion

Opinion: The Morris ALS Principles: A model for empowering all disease communities

27.07.2022

The FDA’s decision to delay its highly anticipated review of AMX0035, a new drug for treating amyotrophic lateral sclerosis, until September highlights an ongoing rift between the needs of people …

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First Opinion

Opinion: There’s no ‘Moonshot’ or ‘Warp Speed’ for rare diseases. There should be

22.07.2022

The term “rare disease” is both an apt descriptor and a misnomer. Individually, each rare disease affects a relatively small number of people. But taken together, more than 30 million …

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Biotech

FDA advisers unanimously endorse Bluebird’s gene therapy for rare disorder

10.06.2022

Advisers to the Food and Drug Administration voted unanimously in favor of approving an investigational gene therapy from Bluebird Bio on Thursday, concluding that its benefits for children with a …

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