The Bettuchi's Amanda Bettuchi and her husband have 3 children. Their middle child, Shane, was born with severe combined immunodeficiency (SCID). Essentially, this means he was born with practically no…
According to a story from Healio, new guidelines for the treatment of different forms of vasculitis recommend the use of biologic therapies as a first-line option for certain patients. It…
The development of treatments and medicines for diseases can take a long time. These treatments come from many hours of research, studies, trials, and waiting for approval from the…
According to a story from Neurology Advisor, the results of a recent study suggest that the presence of psychiatric issues in patients with idiopathic intracranial hypertension is connected to a…
The most common inherited bleeding disorder, von Willebrand disease, appears more likely to cause problems for juvenile males living with the disease than juvenile females, according to a study…
A press release from the US Food and Drug Administration (FDA) is announcing the approval of a new treatment for anemia in adult patients with the rare blood disorder beta…
Happy Friday, readers! We hope everyone's staying warm! This week we're highlighting a new patient story from a rare disease parent in the Czech Republic. Next, we have ann article…
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…
East Anglia's Children's Hospices in Norfolk, U.K., has recently opened up its new facilities after renovations. To celebrate this opening, Kate Middleton, the Duchess of Cambridge, visited and spoke…
According to a story from dailypost.co.uk, Richard Mason, the millionaire founder of moneysupermarket.com, and and baker Alison Faire's daughters Annabelle (aged 4 years) and Imogen (aged 7 years) don't have…
According to a story from Globe Newswire, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced the results of a phase 3 clinical trial that was testing its experimental product candidate…
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 Healio, a recent presentation featured at the FH Foundation Global Summit was a stark reminder of the need for improved treatment for patients with familial…
Copyscape score: 4% A common symptom of Rett Syndrome is apnea, which is a difficulty in breathing. This apnea has been a symptom that has had no treatment for a…
According to a story from pharmaphorum.com, the drug company Genfit has recently released data from a study which was testing the effectiveness of a new approach for the diagnosis of…
According to a story from Medscape, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recently issued a positive recommendation for the drug osilodrostat. This is…
There have been extreme improvements in genetic engineering over the past fifty years since it was pioneered in the 1970s. It has been used for the treatment of various…
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…
According to a story from BioSpace, the gene therapy companies Sarepta Therapeutics, Inc. and StrideBio, Inc. have recently announced the completion of a licensing and collaboration agreement that could lead…
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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…
Cigarettes have been repackaged for the 21st century. The new millennial pass-time is vaping, an electronic device that heats a liquefied solution of nicotine or THC that becomes vaporized as…
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…
According to a story from finanznachricten.de, the drug company Novartis recently announced that the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive…
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