Remove Doctors Remove FDA Approval Remove Genetics Remove Protein
article thumbnail

AI tool may help doctors select best drugs for COVID patients

pharmaphorum

Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients. The post AI tool may help doctors select best drugs for COVID patients appeared first on.

Doctors 135
article thumbnail

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.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Roctavian Becomes First Gene Therapy for Severe Hemophilia A to Get FDA Nod

XTalks

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. The approval came after a couple of setbacks, including a rejection in 2020 and a delay earlier this year.

article thumbnail

How to advance AAV-based gene therapies

Drug Discovery World

As such, they offer highly stable expression of human genes, mRNA, and proteins. in Cell Biology and Biochemistry from University College London and was a post-doctoral fellow at Albert Einstein College of Medicine in New York City. Food & Drug Administration (2022) FDA Approves First Gene Therapy to Treat Adults with Hemophilia B.

article thumbnail

Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

XTalks

SMA is a rare genetic condition that leads to a loss of motor neurons that results in progressive muscle weakness and wasting, paralysis and, when left untreated in its most severe form, breathing difficulties leading to permanent ventilation or death for most patients by the age of two. With a price tag of over $2.5

article thumbnail

STAT+: Foundation charges cancer patients $83,000 for unproven but promising experimental drug

STAT News

But they said the sum covers only the direct costs of designing and manufacturing the vaccines ― which are tailored to the unique genetic makeup of each patient’s tumor ― and that the FDA has approved the breakdown of expenses. It takes at least four months to create the vaccines, according to JLF.

article thumbnail

The democratisation of cell and gene therapy

Drug Discovery World

He says: “What we are seeing from a molecular biology perspective is that the genetic material that we want to pack into viral vectors is becoming increasingly complicated. We are now looking at larger plasmid DNA sequences to carry more genetic material, and so we can get a more delicate endpoint with a much larger plasmid DNA sequence.