Between August 29 and September 1, researchers joined in at ESC 2020 - The Digital Experience, developed and organized by the European Society of Cardiology (ESC). During this online conference,…
To determine the safety and efficacy of experimental gene therapy ST-920, researchers first examined the impact of the drug on mouse models of Fabry disease. As reported in Fabry Disease…
Early this week, biopharmaceutical company Avidity Biosciences announced its inclusion into a collaborative natural history study on myotonic dystrophy type 1 (DM1). The study, END-DM1, is run by the Myotonic…
As reported in Biospace, the life sciences company Revive Therapeutics Ltd. has announced their intention to join a Clinical Trial Agreement (CTA) with the Board of Regents of the University of…
According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals, Inc. has announced recently that its experimental diagnostic 64Cu-SARTATE™ has earned Rare Pediatric Disease designation from the US Food…
To determine disease progression and severity, researchers often use biomarkers, a measurable substance that indicates disease state. According to Myasthenia Gravis News, a new study shows that acetylcholine receptor (AChR)…
Ionis Pharmaceuticals has just released their findings from a study investigating the effect of IONIS-PKK-LRx and IONIS-PKKRx in hereditary angioedema (HAE) patients with different forms of the disease. These therapies work to reduce…
A new study published in the journal Rheumatology and Therapy examined the effect of baricitinib on Rheumatoid Arthritis (RA) patients who have had an inadequate to their methotrexate therapy. Methotrexate and Baricitinib…
Researchers in Canada have just released positive results from their heterozygous familial hypercholesterolemia (HeFH) clinical trial. This investigation was examining the effect of Repatha on teenagers with the condition. The trial…
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…
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