Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
This past week, Target RWE announced data from its TARGET-NASH study, designed to better understand differences in health outcomes for patients with nonalcoholic fatty liver disease (NAFLD). According to their…
According to a story from BioSpace, the drug company Plus Therapeutics has recently announced that its investigational therapy Rhenium NanoLiposomes (RNL™) has earned Orphan Drug designation from the US Food…
Happy Friday! Sept. 13-19 is Balance Awareness Week. It's intended to bring attention to balance-related vestibular disorders such as Meniere’s disease and vestibular migraine. Looking for ways to help out?…
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,…
Bethany Buettner had started coughing...a lot. She had recently faced pneumonia, but after a course of antibiotics she appeared to have recovered...except for her persistent cough. It wasn't long before…
According to a story from Pharmaceutical Business Review, the pharmaceutical company Bayer has recently announced encouraging findings from its phase 4 clinical study. This trial was testing the drug riociguat…
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…
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…
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…
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…
Happy Friday! Monday, Sept. 7 is Duchenne Muscular Dystrophy Awareness Day. DMD is characterized by the inability to make the protein dystrophin in muscles and can result in heart and…
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