What Types of New Therapies are Available for Cystic Fibrosis?
Discussion Cystic fibrosis (CF) was identified in 1938 by Dr. Dorothy Andersen who described 49 patients with pancreatic insufficiency. Since that time significant achievements in the knowledge about the disease and treatments for patients have changed the mortality from a few months to patients living into middle adulthood or even later. Quality of life for patients and their families and friends has also markedly improved. CF is the classic Mendelian autosomal recessive genetic disorder which is a worldwide disorder but affects people of north European ancestry more often where the main mutation is more common. There are more than 2000 variants, but the main mutation is known as Phe508del or F508del. This codes for the cystic fibrosis transmembrane conductance regulator gene (CFTR). Simplistically the CFTR codes for a protein that leaves the endoplasmic reticululum and attaches to the chloride and anion channel at the apical cell membrane which facilitates anion transport out of the cell. This regulates chloride, bicarbonate (needed for respiratory cell protection and pancreatic enzyme activation) and maintains epithelial surface hydration. In countries with a high prevalence, newborn screening provides most of the diagnoses. Location specific protocols can include testing for immunoreactive trypsinogen, pancreatitis-associated protein testing and DNA mutation analysis. Newborn screening is a screening test and a sweat chloride test is considered the gold standard for diagn...
Mississippi health officials have issued an advisory that COVID-19 vaccines are safe, effective and recommended to pregnant women after reports some were turned away at clinics.
AbstractAcute myeloid leukemia (AML) relapse after allogeneic stem cell transplant (alloSCT) remains a major therapeutic challenge. While patients with longer remission after initial alloSCT are recommended to receive cell therapy (CT) such as 2ndalloSCT or donor lymphocyte infusion (DLI), survival for patients who relapse within 6 months of alloSCT has been dismal. We evaluated the outcomes of AML relapse after alloSCT to assess the impact of different treatments on long-term survival. One hundred and seventy-two patients with AML underwent alloSCT at the Penn State Cancer Institute from January 2014 to August 2019....
Patients who have rare retinal diseases — as well as those with more common ones — may benefit from a surge in gene therapy research.Medscape Medical News
Growing interest in liver transplantation to treat nonresectable colorectal liver metastases prompted an international working group to develop consensus guidelines.Reuters Health Information
Incidence of COPD - a group of lung diseases that cause breathing difficulties - in England was down 51 per cent in 2020 compared to 2019, a damning Department of Health report found.
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