The question of whether to go back to school amidst Covid-19 has been a debate across the country. Many universities have seen surges in cases, many involving students in Greek…
Dr. Lisa Sammaritano from Weill Cornell Medical College has explained a way to lower risks within pregnancy for those with rheumatic diseases. Pregnancy and Rheumatic Diseases Patients who have a…
Recently, the Janssen Pharmaceutical Companies of Johnson & Johnson reported that they submitted a New Drug Application (NDA) for intravenously administered UPTRAVI (selexipag). The therapy is designed to treat…
Acknowledgment: This story is sponsored by Chiasma, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
Vertex Pharmaceuticals will present data on the long-term use of their cystic fibrosis (CF) treatments at the 43rd European Cystic Fibrosis Digital Conference, and then again at the 2020 North…
APT-1011 is a treatment for eosinophilic esophagitis, a condition that currently faces an unmet medical need. The medication was originally developed by TPG Capital, but they created a new company,…
Recently, twenty-two year old Jordan Ray of Wellington, Florida spoke with a reporter from The Palm Beach Post, recounting how her life changed in April 2015 during a softball…
by Danielle Bradshaw from In The Cloud Copy There was a case report (called Anti-MUSK positive myasthenia gravis with antiLrp-4 and antigen antibodies) released recently that talked about a patient…
Researchers have announced their identification of a specific gene mutation that can lead to atypical hemolytic uremic syndrome (aHUS). This gene is called DGKE and it is not a part of…
A research team at Ghent University in collaboration with Imec recently released preliminary data on their artificial iris. They are calling it a "smart contact lens." By embedding microelectronics into…
Misophonia is a rare condition that causes patients to have a severe and involuntary reaction to specific sounds. The cause of the condition is still unknown. A challenge misophonia patients face…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Today was the end of the…
Immune-One Therapeutics has just announced that they have dosed their first acute myeloid leukemia (AML) patient in their Phase 1 trial investigating IO-202. This therapy is an immune inhibitory receptor that works…
by Danielle Bradshaw from In The Cloud Copy Research into the common but rare TP53 mutation - which can be found in many Ashkenazi Jewish people - revealed that it…
Just last week, biopharmaceutical company Mustang Bio announced Orphan Drug designation for its therapeutic candidate, MB-207. This unique lentiviral gene therapy is designed for previously treated patients with X-linked…
According to an article from Charcot-Marie-Tooth News, a study was conducted that discovered three new mutations on the GJB1 gene that result in X-linked Charcot-Marie-Tooth disease (CMTX1). The study, which…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler A few weeks ago, during one…
BioMarin has been working to develop treatments for phenylketonuria for 15 years, and it is a cause they are very committed to. That is why they are so excited to…
Lupus Canada and the Lupus Foundation of America have joined together to award the 2020 Lupus Canada Catalyst Award. This grant is intended to fund one year of research that…
When children have problems at school, it is often linked to something bigger going on in another aspect of their life, whether that is at home or with friends. A…
Recently, researchers sought to better understand the natural history and natural progression of type III spinal muscular atrophy (SMA). According to the AJMC, this study offers more insight into…
Several decades ago, gene therapy seemed to be on target to treat a disease by simply replacing a defective gene with a healthy one. A recent article republished in APNews…
On September 25, biotechnology company Vertex Pharmaceuticals Incorporated ("Vertex") announced FDA-approval of KALYDECO (ivacaftor) for pediatric patients with cystic fibrosis (CF). Specifically, KALYDECO can treat infants between 4-6 months…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler When faced with your own mortality,…
by Danielle Bradshaw from In The Cloud Copy Fulcrum Therapeutics, a company that focuses on clinical-stage biopharmaceuticals to improve the quality of life for patients that have rare genetic diseases,…
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