A new genetic test for keratoconus as well as other types of corneal dystrophy was released late last year. The test was created by Avellino and can identify biomarkers of disease. It also looks…
Rocket Pharmaceuticals has recently dosed the first patient in their trial of RP-A501, a treatment for Danon disease. According to BioSpace, this trial will test the safety, tolerability, and efficacy…
Recently, biopharmaceutical company Albireo Pharma announced positive results from a Phase 3 clinical trial. The trial, PEDFIC 1, studied the safety, efficacy, and tolerability of odevixibat for patients with progressive…
Research was presented at The Digital International Liver Congress that proved nidufexor is a safe and viable treatment for nonalcoholic steatohepatitis (NASH), according to Healio. It was shown to reduce body…
According to Parkinson's News Today, Novant Health will soon become the first provider in the Carolinas to offer MR-guided focused ultrasound (MRgFUS). The therapy, with a surgery-like technique, has no…
The CureGRIN Foundation has recently announced that its annual conference will be held virtually this year as a result of the ongoing coronavirus/COVID-19 pandemic. This year's event will be held…
According to Charcot-Marie-Tooth News, the EveryLife Foundation has began a scholarship fund to help rare disease patients in the United States. Living with a rare disease often brings challenges, many…
Elliot Preddie, age 12, is ready to get back to his favorite sport, football. He had to put off playing due to sickle cell disease (SCD). Elliot substituted taekwondo and…
Health Canada has recently granted market authorization to Hoffman La-Roche Limited for their neuromyelitis optica spectrum disorder treatment, ENSPRYNG. Canada is the first country to offer this medication, and hopefully…
Praise Aki Amedal, a Nigerian living in the city of Oriade, has just completed his first book entitled The Biological Prisoner . . . Finding Hope in a Failing Body.…
by Lauren Taylor from In The Cloud Copy Nearly all babies born in the United States today are tested for a certain panel of genetic disorders within 48 hours of…
by Natalie Homan from In The Cloud Copy A review article published in Endocrinology in August of this year highlights the fact that a female's risk of developing nonalcoholic fatty…
by Lauren Taylor from In The Cloud Copy Long QT syndrome, also called LQTS for short, is a disorder of the heart’s rhythm that can possibly cause the heart to…
In recent years, gene therapy has expanded to enormous heights. This experimental technique allows scientists to add, delete, edit, or otherwise use genes to address genetic malfunctions and disorders. Now,…
by Danielle Bradshaw from In The Cloud Copy Throughout the duration of the pandemic, it’s become abundantly clear that scientists still don't have a solid grasp on the diverse array…
by Natalie Homan from In The Cloud Copy In July of this year, doctors in Switzerland started using a new software program that will help them and many others more…
Recently, the MDS Foundation announced that two TP53 gene mutations result in worse patient outcomes in myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). According to their international and multi center study,…
Dayton Abbott is like many kindergarteners; she loves to tell jokes, play games, and hang out with friends during recess. But her playground at Alexander Elementary School wasn't built for…
by Lauren Taylor from In The Cloud Copy As the world continues to watch the effects of COVID-19 sweep around the world, many worry about getting infected. A study from…
Earlier this week, the FDA approved oral azacitidine (Onureg) for patients with acute myeloid leukemia (AML). According to the ASCO Post, Onureg continuously treats adult patients who previously achieved remission.…
This month is Charcot-Marie-Tooth (CMT) Awareness Month. Due to COVID-19, there cannot be any of the typical events, but that doesn't mean there's nothing you can do. From a virtual…
by Lauren Taylor from In The Cloud Copy Rheumatoid arthritis (RA) is an autoimmune and inflammatory disease, which means that your body's healthy cells are attacked by your body’s own…
According to BioSpace, a Phase III trial of Farxiga has produced very positive results. This chronic kidney disease (CKD) treatment was able to dramatically prolong survival in those with stages…
by Lauren Taylor from In The Cloud Copy Autoimmune uveitis is a condition in which abnormal activation of the immune cells takes place within the eye, ultimately leading to the…
by Lauren Taylor from In The Cloud Copy After completing an internship at the world-renowned Cleveland Clinic, one Ohio teenager is now working on changing the world for dementia patients…
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