The GRIN Virtual Conference September 12, 2020 Don't miss the CureGRIN Foundation's GRIN Virtual Conference. The event will be packed with valuable information for the GRIN disorder patient community. Participants…
Caroline Wozniacki has led an interesting life. Born in Denmark, she was immediately thrust into athleticism. Her father was a former football player (that's soccer for you Americans) and her…
Last month, biotechnology company BioMarin sought approval for an investigational gene therapy (valoctocogene roxaparvovec) for patients with hemophilia A. However, their approval was denied. Instead, the company was told that…
Cornell University and reVision Therapeutics have come together through a licensing agreement to develop a treatment for Stargardt disease, according to BioSpace. They will be working on and commercializing Cornell's…
According to a recent article in MedicalXpress, researchers in Japan led by Dr. Shinsuke Ishigaki and his team at Nagoya University, have identified new molecular details about tau’s activity. Alzheimer’s,…
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…
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…
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…
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 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…
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…
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…
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 Having a child, especially for the first time, comes with so many unknowns and questions. What stroller to buy, what to name…
Recently, biopharmaceutical company Fortress Biotech announced positive clinical results for CUTX-101 (copper histidinate). The data highlights the efficacy of CUTX-101 for patients with Menkes disease. As there are currently no…
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