According to a story from BioSpace, the biopharmaceutical company BeiGene Co., Ltd has recently announced that its drug zanubrutinib (marketed as BRUKINSA) has earned Accelerated Approval from the US Food…
For people with sickle cell disease, a common, painful symptom is vaso-occlusive crisis, which is when blood circulation is blocked by sickled red blood cells. This restriction of circulation results…
When your child is constantly at risk of hypoglycemia and their health and life is threatened in the case of any major problem, you have no other choice but to…
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The Golden Age of Health and Wellness is Almost Here A recent article in the Vancouver Weekly News describes the euphoria that accompanied the official declaration of the completion…
For a long time, Huntington's Disease has been monitored and studied through the examination of subjective measures, such as Unified Huntington Disease Rating Scale (UHDRS), and survey-based questions. MC10 is…
According to a story from wptv.com, young mother Sierra Henderson, whose first child is just 18 months old, reported to an urgent care facility recently with some unusual symptoms: a…
The family of a little girl who is battling health problems after suffering a brain tumour are appealing for people to make her birthday extra special by sending her cards…
Researchers at the University of Pennsylvania are working on a new potential gene therapy for Duchenne muscular dystrophy (DMD). DMD is a rare form of muscular dystrophy that primarily affects…
According to a story from marketscreener.com, the biopharmaceutical company Anavex Life Sciences Corp. has announced that its experimental product candidate ANAVEX2-73 has earned Rare Pediatric Disease designation from the US…
WHIM Syndrome WHIM syndrome is a form of primary immunodeficiency. It is caused by a mutation in the CXCR4 gene. WHIM stands for Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. Throughout their…
By Jodee Redmond from In the Cloud Copy The results of a recent study published in Pompe Disease News suggest that a molecular “tag” used to improve the distribution of…
By Rachel Whetstone from In the Cloud Copy Although rare diseases are uncommon by definition, a new study shows that the total number of people affected by rare diseases is…
Modern medication has the ability to save lives and improve living standards. However, medicine is less beneficial when patients’ prescribed treatments are delayed, and entirely ineffective when patients forgo treatment…
Teamwork prescribed to tackle major demographic trends in vascular disease 13th November 2019 207 Marc Bonaca addresses delegates at VIVA 2019 “Caring for vascular patients is going to take a…
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“Myasthenia” means “muscular weakness”. According to a recent article in The Star, studies in Asia indicate that there are approximately five new cases of myasthenia gravis (MG) reported each…
According to a story from pm360online.com, the biopharmaceutical company miRagen Therapeutics, Inc., recently presented Phase I clinical trial data regarding the company's experimental drug cobomarsen. This data was presented at…
Patients in the ICU are under constant surveillance, with consistent monitoring of their blood pressure, blood cultures, and other laboratory markers. These things help doctors understand how the patient is…
Gracie Mellalieu, a twelve-year-old girl from Wales, is excited to headline the Christmas concert at St. Asaph Cathedral. This accomplishment comes after Gracie gained attention for a thank you song…
Over the course of the past nineteen years, scientists were able to successfully map the entirety of the human genome and there have been great advances in genetic testing. These…
Cancer tumors need an abundance of different nutrients to grow. Therefore it would make sense that by cutting off the food supply to cancer tumors, we could successfully inhibit their…
A New Study A recent study published in Nature Immunology and conducted by researchers at the University of Basel has produced influential results regarding primary immunodeficiency disorders (PID). These rare disorders…
Gina, a 14-year-old Jewish girl, had familial dysautonomia when she passed away. She was described as a having a 'positive and upbeat attitude' and enjoyed spending time with her older…
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All parents have fears for their children, but few of them expect their child to be diagnosed with a disease so rare there are only 2-4 diagnoses for every 100,000…
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Marten Devlieger’s sister Karen, a cystic fibrosis patient, died of the disease at the age of thirty-three. According to a recent article in CTV News Canada, Marten was also diagnosed…
Maddie Brown Brush is famous for being on TLC's Sister Wives, and she welcomed her daughter, Evangalynn Kodi, into the world with her husband on August 20. Brown Brush described…
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