According to a story from prnewswire.com, the Chiesi Global Rare Diseases and the biopharmaceutical company ProTalix Biotherapeutics have recently announced that they have submitted a Biologics License Application (BLA) for…
Continue Reading Fabry Disease: A Biologics License Application for Pegunigalsidase Alfa has Been Submitted to the FDA
By Danielle Bradshaw from In The Cloud Copy The cells of hemophilia A patients were taken and genetically modified so that they would create an active clotting factor VIII (or…
Continue Reading Mouse Model of Gene Therapy is Shown to be Effective Against Hemophilia A
Women can do anything that men can do, and vice versa. Well, while that sentiment may be true in our everyday life, it isn't quite true on a genetic…
Continue Reading C4 Genes as a Double-Edged Sword: Sex-Based Differences in Lupus, Schizophrenia, and Sjögren’s Syndrome
IBDsmart Researchers at the University of Otago, led by Michael Shultz, have developed a new app called IBDsmart to help patients with ulcerative colitis or Crohn's disease receive care remotely.…
Continue Reading An App in New Zealand Allows IBD Patients to Communicate with Their Specialists Virtually
According to a story from Pulmonology Advisor, a recent study has evaluated risk factors for the appearance of bronchiectasis in patients with aspirin-exacerbated respiratory disease (AERD). A small number of…
Continue Reading Study Identifies Bronchiectasis Risk Factors in Aspirin-Exacerbated Respiratory Disease
For the last few months, we've been inundated with stories of COVID-19, and with good reason, considering the global pandemic reached over 5 million cases worldwide as of this…
Continue Reading 120 U.K. Patients with Cystic Fibrosis Receive Lung Monitoring Devices
Being at peace with ourselves and our lives makes daily living so much easier. Of course, sometimes it's easier said than done. But one tactic may help improve comfort, confidence,…
Continue Reading A New and Beneficial Tactic for People with Multiple Sclerosis? Mindfulness Training
Passage Bio has been granted the Rare Pediatric Disease designation for their GM1 gangliosidosis treatment, PBGM01. This designation means that Passage will receive a priority review voucher, and it is…
Continue Reading GM1 Gangliosidosis Drug Receives Rare Pediatric Disease Designation
Jalee Pelissier lives with muscular dystrophy. When she was just 13 she became an advocate for others with disorders or disabilities like her own. Now 20, her advocacy has only…
Continue Reading This Muscular Dystrophy Patient has Spent Her Life Working to Improve the Lives of Others
For the past five years, it has become more and more apparent that some people have a greater predisposition to suffer from…
Continue Reading What Causes Dysautonomia After a Concussion?
After six decades of attempting to solve chronic organ rejection, researchers at Houston’s Methodist Hospital and Pittsburgh University have met the challenge. According to an article in Medical Xpress,…
Continue Reading This Breakthrough Finding Could put an End to Chronic Organ Rejection