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STAT+: A young boy’s nightmare diagnosis, and the $3 million one-time treatment that will likely save his life

STAT News

When the long-awaited moment arrived, a nurse helped Adam Hess loosen a tiny plastic clamp on an intravenous line leading to the chest of his son, who lay asleep in a bed at Boston Children’s Hospital. They had been modified in a lab by adding a functional gene to compensate for a defective one.

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Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

XTalks

DEB is a genetic disorder characterized by very fragile skin that rips and blisters easily even from minor friction (like rubbing or scratching) or injury, resulting in open wounds that are prone to skin infections and fibrosis. Vyjuvek is also the first drug approved to treat the disease and is Krystal’s first approved product.

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23andMe makes $400m telehealth play, gulping down Lemonaid

pharmaphorum

The company is best known for its saliva-based health and ancestry DNA testing targeted at consumers, but also uses its genetic database to look for new targets for drug development. The transaction is expected to close before the end of the year.

DNA 104
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Intouch’s Top Lessons From SXSW 2022, Part I

Intouch Solutions

What this looks like in our work: How does a brand compete for mindshare competition, where a patient may listen to their doctor for 10 minutes, but then spends hours scrolling social media for information that may be misinformation or disinformation? It needs to be done with doctors, nurses, and patients.”

Doctors 52
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Building a better future for people with rare diseases in all four UK nations

pharmaphorum

Chaired by the Genetic Alliance, the virtual meeting saw representatives from Scotland, England, Wales, and Northern Ireland discuss the implementation of the UK Government’s Rare Disease Framework. We now know that 80% of rare diseases have a genetic origin. Genomic technology, therefore, has a key role to play in our work,” he said.

Genome 89
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Economist Impact: World Cancer Series – pharmaphorum in attendance, day one (part ii)

pharmaphorum

This is why the Maltese Government has set up ‘nurse navigators’, an innovative cancer care service that “brings a personalised, integrated approach to care both during and after treatment”, according to the World Health Organization (WHO). The Maltese situation and nurse navigators. These have to be tackled, Elliott said.

Nurses 87
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Keeping tabs on Covid-19: Iceni Diagnostics creates new virus detection device and the RDIF announce its vaccine is more than 95 per cent efficient

The Pharma Data

The hand-held testing device uses lateral flow – like a home pregnancy test – to detect the virus’s interaction with sugars around human cells, so that nurses, doctors and patients can all easily perform the test without any prior training and at any location. .