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…
.png)
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,…
The Patient-Centered Outcomes Research Institute (PCORI) has announced that it will be contributing funding for four new studies focused on four different rare diseases. The funding will top out at…
Genetec Engineering and Biotechnology News recently carried a story giving credit to Baylor College researchers for finding microbes living in the gut that are associated with specific symptoms of neurological…
Over the last year, news of COVID-19 has dominated the headlines. Caused by SARS-CoV-2, a coronavirus, the global pandemic is now associated with 116 million diagnoses worldwide and 2.57 million…
A person’s true feelings are often difficult to express during an interview. When asked a question by the interviewer there is no time to rehearse but only grasp at the…
Science 37 and Xperiome have entered into a partnership in order to allow for more viable rare disease research, according to an article in PR Newswire. If the partnership goes…
At first, everything in Amberlyn Smith's pregnancy seemed to be going smoothly. When she was just over 29 weeks pregnant, her screening tests appeared to be normal. However, at…
Congress has recently introduced a new bill that would have a significant impact on the rare disease community if it passes. The legislation, called the Speeding Therapy Access Today (STAT)…
A report, commissioned by the National Organization for Rare Disorders and released by IQVIA, has revealed that the majority of orphan drugs, 79%, treat only rare diseases. Despite this fact,…
According to a story from bloomberg.com, the biopharmaceutical company Rallybio has announced two development programs for therapeutics intended to treat rare diseases. The two programs consist of RLBY212, which is…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
