Unfortunately, there are downsides to a lot of treatments for various diseases, such as price, frequency of doses, and discomfort. Any one of these factors can discourage people from…
According to a story from People, Will Yun Lee plays Dr. Alex Park on the medical drama television series The Good Doctor. Although he isn't a doctor in reality, Mr. Lee has…
According to a story from Soweto Urban, mother Lebohang Leuta is no stranger to the challenges that come with raising and taking care of a child with a chronic rare…
According to a story from EurekAlert!, a collaborative study headed by researchers from Yale University has furthered the understanding of the mechanisms and progression of fibrosis (scarring) in patients with…
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An article appeared recently in BioNews announcing the first-ever approval of an RNA-based therapy for use on the British National Health Service. The name of the drug is Patisiran…
According to a story from The Squamish Chief, mother Emily Tomlinson is amazed by the capabilities of her three year old son Nash Smillie. Nash was born with a severe…
Scientists have been studying animals in order to better understand certain diseases for years. In fact, a genetic disorder that causes blindness called Leber's congenital amaurosis was found in…
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…
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…
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…
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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…
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…
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…
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…
Porphyria is a rare blood disease, with less than 20,000 cases in the U.S. every year. Because of this rarity, many people are unaware that the symptoms they experience have…
The Bio Patient and Health Advocacy Summit of 2019 was recently held in Washington D.C. One of the panels addressed patient engagement with the FDA and how patients, advocates, families, and organizations…
Web-based Rare Chronic Illness Support Group 2nd Thursday of each month This is a peer support group for patients living with chronic, rare diseases. Featuring Cara Oppegard, MSW intern To…
According to a story from Pulmonary Advisor, the rare condition pulmonary arterial hypertension (PAH) is mostly known for the impacts that it can have on the lungs and on the…
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