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Topical Gene Therapy FDA-Approved for Severe Skin Disease, Dystrophic Epidermolysis Bullosa

PLOS: DNA Science

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. Earlier results were published in Nature Medicine.

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First Blood Test for Alzheimer Disease Available in U.S.

The Pharma Data

30, 2020 — The first blood test to help diagnose Alzheimer disease is now available in the United States, but some experts have a number of concerns about it. Louis is meant for people 60 years and older who have cognitive issues and are being assessed for Alzheimer disease. states and was just approved for sale in Europe.

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New Hope Against Diseases Marked by Progressive Scarring of Lung Tissue

The Pharma Data

13 in the New England Journal of Medicine , involved patients with high blood pressure in the lungs caused by interstitial lung disease (ILD). It can have a range of causes, from smoking, to occupational exposure to toxins like asbestos, as well as autoimmune diseases like rheumatoid arthritis. The study, published online Jan.

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FDA Approves Expanded BOTOX® (onabotulinumtoxinA) Label to Include Eight New Muscles to Treat Adults with Upper Limb Spasticity

The Pharma Data

BOTOX ® was first approved by the FDA in 1989 for two rare eye muscle disorders – blepharospasm and strabismus in adults. Adult Patients treated for overactive bladder due to neurologic disease: In clinical trials, 30.6% Tell your doctor if you have any breathing-related problems. About BOTOX ®.

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FDA Grants Priority Review to Genentech’s Esbriet (pirfenidone) for Unclassifiable Interstitial Lung Disease

The Pharma Data

Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and granted Priority Review for Esbriet ® (pirfenidone) for the treatment of unclassifiable interstitial lung disease (UILD). The FDA is expected to make a decision on approval by May 2021. Since its U.S. Esbriet U.S.

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U.S. FDA Accepts AbbVie’s New Drug Application for Atogepant for the Preventive Treatment of Migraine

The Pharma Data

– Migraine is a debilitating neurological disease affecting 39 million people in the U.S.[2] Migraine is a complex, chronic disease with attacks that are often incapacitating and can include headache pain as well as neurologic and autonomic symptoms. AbbVie anticipates a regulatory decision in late Q3 2021.

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FDA OKs First Over-the-Counter Home Test for COVID-19

The Pharma Data

I hope that this authorization is just the beginning and will lead to more at-home diagnostics for COVID and other infectious diseases.” ” Last week, the FDA approved a different at-home test, but it requires samples to be mailed to a lab to get the results.

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