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Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical genetherapy 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 genetherapy.
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.
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.
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.
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.
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 genetherapy. The training challenge.
Because we knew that Orphan Medicines, led by the genetherapy 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.
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