As Patient Worthy readers, we hope you and your families are doing well during this challenging time. The health and safety of our rare community is always at the…
Results of one of the largest and most important studies in a decade, the International Study of Comparative Health Effectiveness with Medical and Invasive Approaches (ISCHEMIA), were just announced.…
While many patients are grateful for their doctors and treatments, how many know why they are so passionate about what they do? In an interview with Dr. Patrick Flume,…
Hi everyone, Another week of life in COVID-19 era has passed! Today we have an article on a potential Coronavirus safety update. Next, we have the release of a new…
Researchers at Columbia University have discovered a new gene, TBK1, that is linked to the progression of amyotrophic lateral sclerosis (ALS). A lack of understanding of a rare disease hinders…
Michael and Armené Kapamajian were at home in Los Angeles settling in with their one week old baby, Sasoun, when their doctor called and asked them to come in…
According to a release on Public News Service, the state of West Virginia just implemented a new bipartisan law to support individuals with rare diseases and their families, a particular…
According to a story from BioSpace, Acceleron Pharma Inc. and Bristol Myers Squibb have announced recently that the US Food and Drug Administration (FDA) has recently approved a new drug…
April 17th, 2020 is World Hemophilia Day. This event is held to help foster awareness about hemophilia and other related bleeding disorders. It is a day when this rare community…
April 22nd, 2020 heralds the beginning of World Primary Immunodeficiency (PI) Week. This will be the ten year anniversary of the event, which is designed to spread awareness about PI…
April 15th, 2020 will be the 7th annual International Pompe Day, a day of recognition and fostering awareness around Pompe disease organized by the International Pompe Association. Like many rare…
In this time of cancellations, closures, and shutdowns, many companies and individuals have found unique and creative ways to shift their services online. Better yet, some have temporarily waved their…
A recent study published in the Orphanet Journal of Rare Diseases examines the psychological burden of mitochondrial diseases in children that are suspected of having some form of it but have yet…
According to a story from Mirror Online, Vera Twomey from Cork, Ireland, was devastated when her four month old daughter had a severe seizure that lasted 45 minutes. Before long,…
According to a story from Hindawi, a recent study focused on congenital central hypoventilation syndrome serves to bring to light the importance of genetic testing of close family members based…
What is Repurposing? Repurposing involves testing whether or not a compound approved of another condition may be capable of also treating an illness with no approved treatment option. It's advantageous,…
Dr. Pierre Fenaux, of Hôpital Saint Louis, Paris and lead author of the MEDALIST trial, told Ash Clinical News that treatment with the investigational drug, luspatercept, improved the rate of…
According to a story from Sleep Review, the drug company Jazz Pharmaceuticals plc has recently filed a New Drug Application (NDA) for its investigational treatment JZP-258, which is being developed…
Emily Knakmuhs's two-and-a-half-year-old Kennedie had just made it past a tough couple of years. When she was born, her family noticed she wasn't growing, but her pediatrician attributed it to…
As originally reported in News Medical, a new study suggests that gastric bypass bariatric surgery may be the most effective treatment for non-alcoholic fatty liver disease (NAFLD). This disorder currently…
Hi everyone, We hope you're having a peaceful quarantine and taking care of yourself. As we all know, updates about COVID-19 is dominating the news right now. Of course, infomation…
According to a story from pharmaphorum.com, Jim Kuhn had a successful career in business for over 30 years, but everything changed a decade ago when he was first diagnosed with…
As originally reported in Fabry Disease News, the potentially fatal genetic disorder Fabry disease can be diagnosed most effectively in infancy by newborn screening programs. By identifying the disease early,…
In a Special Issue of the journal Disease Models and Mechanisms Highlights, James J. Dowling et al describes pediatric neuromuscular diseases as mostly genetic and affecting areas of the peripheral…
Pam Kelley, a reporter whose focus is on the challenges facing people below the poverty level, submitted this story to the Charlotte Magazine. Pam conducted interviews with Cheryl Potts over…
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