What Caused This Summer Birthday Party Rash?
Discussion Panniculitides are a diverse group of problems involving inflammation of subcutaneous fat. Subcutaneous fat has limited ability to show different patterns of disease and therefore the clinical appearance is the same but has different etiologies. The skin appears red or purple, swollen, may have discrete nodule(s) or plaque(s) and the affected area can be painful or pruritic. If necessary skin biopsy can help determine the etiology. Overall panniculitides are not very common but some presentations are expected because of particular exposure (i.e. cold) or inflammatory disease processes. Types of panniculitides in...
Source: PediatricEducation.org - August 22, 2022 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news

Novel Liver-Targeting Drug Offers Hope for AAT Deficiency Novel Liver-Targeting Drug Offers Hope for AAT Deficiency
The investigational drug fazirsiran improved important liver parameters associated with alpha-1-antitrypsin deficiency.Medscape Medical News (Source: Medscape Gastroenterology Headlines)
Source: Medscape Gastroenterology Headlines - June 28, 2022 Category: Gastroenterology Tags: Gastroenterology News Source Type: news

Inhibrx Receives FDA Orphan-Drug Designation for INBRX-101 for the Treatment of Alpha-1 Antitrypsin Deficiency
Interim results reported in October 2021 from the Phase 1 study showed a favorable safety and tolerability profile with no drug-related severe or serious adverse events at doses up to and including 120 mg/kg single dose and 80 mg/kg multi-dose administered... Biopharmaceuticals, FDA Inhibrx, INBRX-101, alpha-1 antitrypsin deficiency (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - March 3, 2022 Category: Pharmaceuticals Source Type: news

What Does Alpha-1-Antitrypsin Do?
Title: What Does Alpha-1-Antitrypsin Do?Category: Diseases and ConditionsCreated: 9/28/2021 12:00:00 AMLast Editorial Review: 9/28/2021 12:00:00 AM (Source: MedicineNet Lungs General)
Source: MedicineNet Lungs General - September 28, 2021 Category: Respiratory Medicine Source Type: news

Diagnosis of genetic condition could help patients stop smoking and prevent lung disease
(RCSI) New research shows that people diagnosed with a genetic condition, called alpha-1 antitrypsin deficiency (AATD), are far more likely to stop smoking and therefore prevent the development of lung disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - March 3, 2021 Category: International Medicine & Public Health Source Type: news

Vertex shuts down trial of liver drug, spurring a selloff
Shares of Vertex Pharmaceuticals fell after-hours on Wednesday, spurring a loss of around $7 billion in market cap, after discontinuing work on one of its next-generation drugs.   The Boston biotech announced it halted work on a potential treatment for a lung and liver disorder called alpha 1 antitrypsin deficiency after patients began experiencing side effects.  JMP Securities analyst Liisa Bayko told the Business Journal earlier this year that the drug was “the firs t shoe to drop” in Vertex’s… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - October 15, 2020 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

What Is the Life Expectancy of Someone With Alpha-1 Antitrypsin Deficiency?
Title: What Is the Life Expectancy of Someone With Alpha-1 Antitrypsin Deficiency?Category: Diseases and ConditionsCreated: 9/29/2020 12:00:00 AMLast Editorial Review: 9/29/2020 12:00:00 AM (Source: MedicineNet Lungs General)
Source: MedicineNet Lungs General - September 29, 2020 Category: Respiratory Medicine Source Type: news

What Does the Liver Do?
Discussion The liver is one of the largest organs in the body, weighing just over 3 pounds in an adult. It is found in the upper right abdomen, under the right dome of the diaphragm. Grossly, it has asymmetric lobes with the right being larger than the left. The lobes are separated by a fibrous connective tissue band that also anchors the liver in the abdominal cavity. The gallbladder is located on the inferior surface of the liver and stores bile, which is then released into the duodenum. Microscopically, the liver cells are arranged in lobules with canals carrying blood vessels and bile ducts. At any moment about 10-13% ...
Source: PediatricEducation.org - April 1, 2019 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

Human protein produced in CHO-cells can save donor blood
(Technical University of Denmark) Researchers from DTU Biosustain have successfully produced mammalian cell lines (CHO) that can produce 1.2 g/L recombinant Alpha-1-antitrypsin proteins with human glycosylation profiles. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - March 27, 2019 Category: Biology Source Type: news

Abundant proteins in platelet-rich fibrin and their potential contribution to wound healing: An explorative proteomics study and review of the literature
ConclusionThe results of this study indicated that PRF abundantly contains blood-origin actors which were previously reported for their direct contribution to wound healing. Further studies exploring the protein content of PRF are needed to reveal its undisclosed potential roles in the healing process. (Source: Dental Technology Blog)
Source: Dental Technology Blog - September 5, 2018 Category: Dentistry Source Type: news

CRISPR genome editing technology can correct alpha-1 antitrypsin deficiency
(Mary Ann Liebert, Inc./Genetic Engineering News) Groundbreaking research demonstrates proof-of-concept for using CRISPR-Cas9 genome editing technology to correct the gene mutation responsible for alpha-1 antitrypsin (AAT) deficiency, successfully making a targeted gene correction in the livers of affected mice that restored at least low levels of normal AAT. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - July 2, 2018 Category: Biology Source Type: news

Updates on new therapies in development for rare liver diseases
(European Association for the Study of the Liver) Long-term data with sebelipase alfa for lysosomal acid lipase deficiency and preliminary data for investigational RNAi therapeutics for acute intermittent porphyria and alpha-1 antitrypsin deficiency add continued hope for the future management of metabolic and rare liver diseases. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 14, 2018 Category: International Medicine & Public Health Source Type: news

Alpha-1 Antitrypsin Deficiency —From Genes to Therapies
NIH funded research has identified genetic factors and clinical manifestations of alpha-1 antitrypsin deficiency which may lead to future treatments. (Source: NIDDK News)
Source: NIDDK News - March 15, 2018 Category: Endocrinology Source Type: news

AATD Liver Disease Differs in Kids, Adults
(MedPage Today) -- Discrepancies seen by age in risk factors for hepatic involvement in alpha-1 antitrypsin deficiency (Source: MedPage Today Gastroenterology)
Source: MedPage Today Gastroenterology - February 22, 2018 Category: Gastroenterology Source Type: news

First John W. Walsh Translational Research Award granted
(Boston University School of Medicine) The Alpha-1 Foundation celebrates the first granting of the John W. Walsh Translational Research Award in Alpha-1 Antitrypsin Deficiency, an honorable award named after John W. Walsh, co-founder of the Alpha-1 Foundation, a visionary man who dedicated his life's work to finding a cure for Alpha-1 Antitrypsin Deficiency and helping those affected by Alpha-1. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 31, 2018 Category: International Medicine & Public Health Source Type: news