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

XTalks

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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One Minute Summary of May 2024 Patient Forum

WCG Clinical

I’m actually a second career nurse. I am in the Doctorate in Nursing Practice program at the University of California, San Francisco, and I’m really looking forward to, becoming the first person in my family on either side to achieve a doctorate degree. He was three and a half at the time.

Nurses 52
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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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Novartis hails digital strategy as profits hold up during pandemic

pharmaphorum

Under the leadership of CEO Vas Narasimhan, who took over in 2018, the company has focused on using digital technology to engage with both patients and doctors. Marie-France Tschudin, president of Novartis’s pharmaceuticals business, said digital technology had helped to improve productivity and its interactions with doctors.

Doctors 111
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Could virtual reality become an essential tool for healthcare education?

pharmaphorum

Due to the pandemic, more people than ever had to turn to digital healthcare to achieve basic activities, such as speaking with a doctor or booking a visit with a healthcare professional. Spark Therapeutics developed a VR product , alongside a partner, to teach haemophilia patients about gene therapy. The training challenge.

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Three pillars of post-pandemic launch excellence

pharmaphorum

Because we knew that Orphan Medicines, led by the gene therapy Zolgensma and the Cystic Fibrosis treatment Trikafta, had in many cases actually outperformed expectations in 2020, we assessed the first six months of sales for New Active Substance launches with and without Orphan medicines.

Sales 95
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Digital health: the new normal?

pharmaphorum

Whether catching up with relatives, attending a business meeting, or even visiting a doctor, it became commonplace to conduct this type of activity via a screen. According a Pew Research study, 81% of participants said they have used video calls to talk to others since the onset of the pandemic. Tapping into data.

Genetics 145