How A Doctor Approaches Care for Patients with Cystic Fibrosis
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,…
Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
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,…
According to BioSpace, an experimental drug designed to treat cancer and HIV may also be poised to treat patients with COVID-19. The drug, called leronlimab, was created by biotechnology…
A news release from Benzinga shares that biotechnology company Emerald Health Pharmaceuticals is beginning a Phase 2 clinical study on EHP-101 as a treatment for patients with systemic sclerosis.…
According to a study summarized in Gastroenterology Consultant, pregnant women with intrahepatic cholestasis of pregnancy are much more likely to also be diagnosed with nonalcoholic fatty liver disease. You can…
As initially covered by MyLondon News, the family of a London boy with spinal muscular atrophy (SMA) is looking to advocate for others with this genetic disorder. Additionally, they…
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…
When some people hear "placebo effect," it conjures up images of sugar pills designed to stimulate patient belief. But according to Healio, the term has a different meaning in…
According to a press release from the Boston Children's Hospital, a study has discovered the efficacy of CRISPR-Cas9 gene-editing technology in the treatment of facioscapulohumeral muscular dystrophy (FSHD). As…
According to a press release on Global Newswire, Novartis has received a positive opinion on Cosentyx® from the Committee for Medicinal Products for Human Use (CHMP). This positive opinion…
Research findings can sometimes come from unexpected places. For example, according to Parkinson's News Today, the benefits and shortcomings of an investigative gene therapy were explored through the post-mortem analysis…
People across the globe are experiencing an unprecedented time of uncertainty as COVID-19 continues to spread. However, this pandemic has also brought about something wonderful: people stepping up to…
As stated in a press release from the University of Illinois at Urbana-Champaign, researchers have discovered a bacterial protein fragment that is destructive to lung tissue. This can rapidly…
According to a press release from FirstWord Pharma, Gen and Alnylam Pharmaceuticals have signed an exclusive distribution deal in Turkey for RNAi therapeutic ONPATTRO. This therapy drug will treat…
Want to learn more about recognizing symptoms and diagnosing patients? Well, now you can. In a MedPage article, authors encourage readers to follow along on a case study to…
"A steak saved my life," writes columnist Damon Young, 41, in his article for GQ, as he shares the story of his diagnosis with Takayasu's arteritis. After over-indulging at dinner,…
In a period of unprecedented changes, it is understandable that people may question what their lives may look like moving forward. COVID-19 has now spread to 196 countries and…
In June 2019, Dicerna Pharmaceuticals submitted a clinical trial application for their DCR-A1AT therapy and expects to treat the first patient with A1AD-related liver disease near the end of…
As previously published in Scientific American, slightly after her first birthday, Emma Larson lost the use of her legs and started experiencing difficulty crawling. She was diagnosed with spinal muscular…
The world is experiencing an unprecedented time in the medical realm with the rapid spread of COVID-19, a novel coronavirus. It may be difficult to understand how coronavirus may impact…
In an interview with Medgadget, Dr. Christy Sheehy and Dr. Zachary Helft of C. Light Technologies discuss up-and-coming technologies in the diagnosis and treatment of neurological diseases such as…
Is it safe for those with autoimmune rheumatic diseases to undergo the termination of a pregnancy? According to research published in Arthritis & Rheumatology, the answer is yes. Learn more…
Researchers from the Murdoch Children’s Research Institute (MCRI) have recently discovered a previously unknown and uncharted neurodegenerative disorder. This rare disorder, which has not yet been named, affects children. It…
Because of the current global situation involving coronavirus (COVID-19), many medical organizations have canceled or postponed their annual meetings. The American Academy of Allergy, Asthma, and Immunology (AAAAI) is one…
Those diagnosed with paraganglioma-pheochromocytoma, squamous cell carcinoma of the skin (cSCC), adrenocortical carcinoma (ACC), and carcinoma of unknown primary (CUP) may soon have an additional option for treatment. According to…
Patients with congenital diseases often experience a range of uncomfortable symptoms. On one side of the world, a family with erythromelalgia, a painful condition which can cause redness and swelling…