Severe Combined Immunodeficiency Severe combined immunodeficiency (SCID) is a group of diseases caused by mutations that affect how the immune system works to fight infections. These anomalies are usually passed…
With genetic disorders, there is always a big question: do specific gene mutations respond differently to treatment? How can treatment be targeted to best suit the needs of patients? According…
The rare liver disease primary sclerosing cholangitis (PSC) may impact just .01% of people, but the extremely pernicious disorder's attacks on the liver mean bearers of the disorder account for…
Each year, researchers make new strides into genetic research, including the discovery of new genetic disorders. According to Medical XPress, researchers from the University of Portsmouth and the University of…
Rare disease patients throughout India have expressed their disappointment and anger towards the country's new policy centered on rare conditions. One of their main concerns is that the policy changes…
On April 2nd, NFL scouts made their way to Eugene, Oregon to check out some of college football's most talented players. They watched as athletes completed the vertical jump, 40-yard…
Cancer Network recently reported that Jazz Pharmaceuticals’ Vyxeos was approved by the FDA to treat therapy-related pediatric patients one year or older who were newly diagnosed with acute myeloid…
Financial Burdens in Rare Disease In 2019, 20% of all adults in the United States have reported that they had large medical bills that were unexpected. 18% currently had medical…
In a recent contribution to MSN, Dr. Christopher Austin, director of the NIH Advancing Translational Sciences, compared the medical community’s record-breaking approach to the COVID-19 pandemic with current efforts…
Macey Brietenback, a resident of Bel Air, Maryland, is nineteen years old and has endured various operating procedures over 70 times. WJZ has been following Macey’s struggle with her…
Rare disease patients face a number of obstacles when it comes to getting the proper diagnosis and treatment. There's a lot of work being done to address these problems, some…
NORD (the National Organization for Rare Disorders), through PR Newswire, recently announced the results of a new study that reveals the total number of generics, orphan products, and biosimilars currently…
MSN News recently interviewed Dr. Laxmikant Palo, with the conversation focusing on the situations of people living with rare diseases in India and how to improve them. According to Dr.…
For years, scientists have known the mechanisms underlying normal, as well as the disease-causing (pathogenic) versions, of prions. But according to a recent article published in Sci-Tech Daily, no one…
Despite the pandemic, there were many strides in the field of medicine and research in 2020. For example, researchers first discovered and identified a rare disease called VEXAS syndrome. The…
No longer the new player on the team, next-generation sequencing (NGS) is well established as having an influence in clinical care. In recent years, NGS has been responsible for many…
Rare disease therapies have an exorbitant cost. Fewer patients means higher costs in order to support the development of treatments. In the United States, 966 billion was spent in 2019…
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Rare diseases are much more common than people think. The word "rare" is off-putting. But the word only pertains to each individual rare condition. Collectively, rare diseases are quite common.…
As the pandemic shifted cocktail hour onto zoom and professors posted their full course material online, many rare patients reveled. Not because we weren’t suffering through the pandemic too, but…
Genomic testing is a great way to learn not just about your genes, but how your genes impact your health. In support of the Phase 2 CRESTONE clinical trial, a…
Rare Disease Day is February 28th. This year, it caused a lot of reflection among healthcare professionals and families impacted by rare conditions. How has the pandemic shifted progress in…
According to a recent article in Fierce Biotech, most cancers progress through constant cell division. Scientists at Vanderbilt University are determined to find the reason for this mysterious cell division.…
Dicaprio Bootle is on a mission: to become a star athlete in the NFL. However, he's preparing to give back along the way to his goal. Dicaprio is fundraising for…
As reported in NewsWise; rare drugs are few and far between, with a cliental of less than 200,000 Americans dissuading drug companies from taking on the enormous charge of developing…
Most people who are unhappy with the price of their medication are not aware of drug prices in the truly high-end segment of the market. GoodRx, the prescription drug watchdog,…
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