According to a story from the Vancouver Sun, five rare disease drugs just received coverage in British Columbia. The funding comes from savings accumulated thanks to a recent agreement that…
According to a story from news-medical.net, a recent study that the reduced expression of miR-125a-5p results in an increase in the concentration of the protein TIMP-1. Prior research has associated…
A new digital platform has been created with hopes to assist cystic fibrosis (CF) patients at the comfort of their own homes, limiting hospital visits, reports Evening Standard. This new…
Originally reported by The Jerusalem Post, many adopted children from Eastern European countries suffer from fetal alcohol spectrum disorder caused by their mothers drinking alcohol excessively when pregnant. Many Israelis families…
According to an article from globenewswire.com, the pharmaceutical company Amicus Therapeutics announced that the Food and Drug Administration (FDA) has the company's New Drug Application for migalastat with filing for…
Sailormen, Inc. represents one of the largest franchise groups of Popeyes Louisiana Kitchen restaurants in the United States. The company includes 136 stores across six states. How does this related…
One of the most impressive moments of childhood is when a young child takes his or her first steps. Natalie Essex, age four, loves to walk. For Natalie, spinal muscular…
According to an article from news-medical.net, a group of researchers from the Telomere and Telomerase Group at the Spanish National Cancer Research Centre have been able to pin down the…
According to an article from Newswise, another recent study is providing further support to the connection between severe gum disease and the development of certain types of cancer. Periodontitis is…
Drugs reports a new study revealing the harmful effects of Zika virus in pregnant monkeys, which might have correlation to the known linkage between Zika and diseases such as microcephaly.…
February is Eisenmenger Syndrome Awareness month. Eisenmenger syndrome is very rare and devastating, so it deserves our attention. Let's get to know a little more about this heart condition. Eisenmenger syndrome…
February 16, 2018 Governor Herbert Declarations: Rare Disease Day on February 28th Undiagnosed Rare Disease Day on April 29th Park City, Utah. (February 16, 2018) – Rare and Undiagnosed Network…
Katie Price, a former British model, has accepted the challenge of running the next London marathon with intent to fundraise for IPF, idiopathic pulmonary fibrosis, a disease her mother was…
According to a story from biomedcentral.com, doctors have begun screening infants for tyrosinemia type 1. However, it is still unclear how accurate the screening process for this genetic disorder is.…
According to a story from Specialty Pharmacy Times, medical professions are starting to recognize the need for a new standardized treatment plan for pediatric rheumatic diseases. Pediatric diseases are illnesses…
Little Natalie Essex was diagnosed with spinal muscular atrophy type 2 (SMA) when she was only a year and a half, reports CBC News. It all started when her mother, Meredith,…
Dangerous forms of brain cancer such as glioblastoma are almost impossible to treat. However, new research by Dr. Roel Verhaak could change that. Glioblastoma is the most aggressive form for…
According to a story from EurekAlert, a recent study published in the journal Pediatric Research reveals new protein biomarkers that researchers can use to evaluate how patients with progeria have reacted…
Happy Friday, Patient Worthians! This week, we have a video from a PKU patient telling a first-hand account of the deterioration and serious consequences he faced after he went astray…
According to a story from biospace.com, a treatment developed by the pharmaceutical company Pfizer was given Breakthrough Therapy designation by the Food and Drug Administration (FDA). The drug is a…
According to an article from news-medical.net, researchers are beginning to delve into the causes of a rare lung disease called primary ciliary dyskinesia. This research is revealing previously unknown information…
The biotech company Myonexus Therapeutics may soon begin clinical trials for muscular dystrophy gene therapy treatment after obtaining nearly $2.5 million of investments. The research behind the new treatment was…
Just this past week 17-year-old Tina McElligott from Kilflynn, Co.Kerry, passed away from rare disease complications, reported Irish Mirror. She had suffered her entire life with Alpers syndrome, having consistent and intense…
Nothing prepares families for the myriad of tests with strange sounding names that are required when they start to look for solutions to the problems brought about by a member's…
Elizabeth Jane Allen, known best as "Betty", will be honored this week for the wonderful woman she was and the lasting impact she has made on the cystic fibrosis community, reports…
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