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Amvuttra RNAi Therapeutic Wins FDA Approval for Rare Genetic Protein Disorder

XTalks

Amvuttra’s FDA approval is based on positive data from the global, randomized, open-label, multicenter HELIOS-A Phase III study from a period of nine months. TTR is made in the liver and is involved in carrying the thyroid hormone thyroxine (T4) and retinol (vitamin A) to the liver.

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COVID-19 and Melatonin: Researchers Use AI To Study New Treatment Option

XTalks

In addition to innovative new antibody drugs and mRNA vaccines, researchers have found that the sleep-regulating hormone melatonin may also offer protection against COVID-19. This approach allowed them to find disease conditions similar to COVID-19 for which approved treatments were available.

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Sanofi snaps up acne vaccine developer Origimm Bio

pharmaphorum

The French group said that ORI-001, a vaccine based on recombinant proteins from the Cutibacterium acnes (formerly Propionibacterium acnes ) bacterium that is often found in acne lesions, could be the first ever vaccine for the condition.

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Ascendis’s SKYTROFA; MicroTransponder’s VNS System (Vivistim); BioMarin’s Voxzogo; Zimmer-Canary’s Smart Knee Implant; COVID-19 impact on Solid Organ Transplants

Delveinsight

FDA’s Green Flag to Ascendis’s Once-Weekly SKYTROFA for Treatment of Pediatric Growth Hormone Deficiency. Growth Hormone Deficiency , commonly known as dwarfism, can constitute short stature and complications with metabolism, which arise due to inadequate production of growth hormones by the pituitary gland inside the body.

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Trodelvy has been Approved for ER+/HER2- Patients with Advanced or Metastatic Breast Cancers

XTalks

The FDA approval is an important step forward for both women and men living with metastatic breast cancer, especially for those individuals whose tumor is no longer responding to endocrine-based therapies and who are facing a poor prognosis,” said Laura Carfang, executive director, SurvivingBreastCancer.org, in the press release.

HR 52
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Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

Symptoms and Etiology: Caused by an overproduction of growth hormone, acromegaly primarily affects adults. By reducing the amount of growth hormone in the blood, progressive enlargement of bones should be slowed. By reducing the amount of growth hormone in the blood, progressive enlargement of bones should be slowed.

Trials 98
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Monthly News Roundup – December 2020

The Pharma Data

The messenger RNA (mRNA) vaccines encode a form of the spike (S) protein of SARS-CoV-2 virus. The vaccine teaches the cells to make a piece of the spike protein, which triggers an immune response to help prevent illness if later exposed to the virus. The partial piece of the spike protein cannot cause COVID-19 disease.