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Tag: rare disease

Biotech

Sarepta wins new drug approval for patients with Duchenne muscular dystrophy, its third marketed treatment

25.02.2021

The Food and Drug Administration on Thursday approved a new medicine from Sarepta Therapeutics that treats certain patients with Duchenne muscular dystrophy. The new drug, called Amondys 45, is Sarepta’s …

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First Opinion / Pharma

Opinion: Ultra-rare but not forgotten: New drug development paradigms to treat the rarest of diseases

17.02.2021

In addition to the most visible effects of Covid-19 — tragic deaths, lingering illness, economic turmoil, and more — the pandemic is affecting much that is out of the spotlight. …

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Biotech / Health / Pharma

Novartis and the Gates Foundation aim to create a more practical gene therapy for sickle cell patients around the world

17.02.2021

Novartis and the Bill and Melinda Gates Foundation are joining forces to discover and develop a gene therapy to cure sickle cell disease with a one-step, one-time treatment that is …

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First Opinion / Pharma

Opinion: The Biden administration needs to look beyond ICER for evaluating drug therapies

09.02.2021

As breakthrough drugs stream out of biopharmaceutical laboratories, how much they should cost and who will get access to them remain thorny issues. During his campaign for president, Joe Biden …

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Coronavirus / Health

‘Frustrated and panicking’: For some rare disease patients, shortages of protective gear pose a continued threat

16.12.2020

When the coronavirus sent cities across the United States into shutdown in March, Laura Bonnell realized that her family’s supply of masks and disinfectant wipes was quickly running out. “We …

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