by Jodee Redmond from In The Cloud Copy Rich Horgan is the founder of the Boston-based biotech non-profit, Cure Rare Diseases. The 28-year-old decided to take action over concerns about…
By Caitlin Seida from In The Cloud Copy If you've never heard of CDG, or congenital disorders of glycosylation, you're not alone. The rare family of diseases, all inheritable, are…
Hi everyone! Times are hard right now, but we hope you and your loved ones are staying safe right now. Today, we're bringing you three stories: first, we have some…
According to BioSpace, healthcare technology company RDMD has raised $14 million in Series A financing, and developed a partnership with UCB. In addition to growing their platform to discuss…
In an interview with Healio, Dr. Jennifer Miller says that there are signs of hope for improved outcomes in patients living with Prader-Willi syndrome, a rare genetic disorder. Dr. Miller…
If you're like me, news about COVID-19 has been dominating your life recently. The novel coronavirus rapidly spread across the world over the past few months. At the time…
Fabry disease brings symptoms that can affect multiple organs throughout the body. The kidneys and gastrointestinal system are commonly affected, which can make life difficult. According to an article from…
According to Medical XPress, researchers have discovered a new potential therapy for patients with pancreatic cancer. By changing the pH balance within cancer cells, researchers could trigger cell death. Read…
Predicting the disease progression of idiopathic pulmonary fibrosis (IPF) is extremely important, as this condition progresses differently depending on the affected individual. Knowing if one has a poor outcome while…
10-year-old Joey Koslowski doesn't let the small...err, the big stuff get him down. According to MyCentralJersey, the boy is "the youngest child to have the largest mass in the world" caused…
According to Parkinson's News, researchers have discovered that specific immune cells in the blood of patients with Parkinson's disease appear up to 10 years before motor skills are impacted. This…
Ibudilast is a drug that has been used to treat asthma, but it is now being evaluated as a treatment for COVID-19. Acute respiratory distress syndrome (ARDS) is a severe…
An earlier online article in JCO Precision Oncology presents a study of next-generation sequencing (NGS) as it applies to molecular profiling of non-gastrointestinal stromal (GIST) soft tissue sarcomas .…
According to a story from Barron's, the biotechnology company Alexion Pharmaceuticals has recently announced plans to test its drug Ultomiris as a potential treatment for COVID-19. As the scientific community…
So you're looking to get pregnant or curious about some part of it. Well, you've come to the right place! If you're looking to have a baby, but you also…
According to a story from The Charlotte Weekly, local resident Kevin Tobin recently ran 400 laps around his house---the equivalent of a marathon---to help raise money for Dan Donoher, a…
Saying "coronavirus changed the world" seems like a bit of an understatement right now. Jobs have embraced "work from home" en masse. Doctor's appointments are being rescheduled, school is online, and most…
According to a recent FDA press release, the agency has given its first approval for a drug developed specifically for adult patients diagnosed with upper tract, low-grade, urothelial cancer.…
Ashlyn Sanders was a graduate student at Duke University when she experienced her first seizure. The UNC alumna was shocked. She pursued an MRI, which ended up discovering a…
George and Josie Taylor fell in love in their 20s. 52 years later and happily married, the couple believed that they could conquer anything together. But COVID-19 threw a…
According to the UN News, the first ever World Chagas Disease Day was celebrated on April 14, 2020 to raise awareness for Chagas Disease, a rare disease spread by…
According to Ankylosing Spondylitis News, a recent study has found that TNF inhibitors are often not effective treatments for patients with ankylosing spondylitis. Read the full study in BMC Rheumatology.…
According to Medical XPress, Australian researchers have discovered a new and more effective way to detect remaining cancerous tissue in patients with breast cancer during surgery. This technique will assist…
In a post on the Metachromatic Leukodystrophy (MLD) Foundation's website, the organization discusses the possibilities of using gene therapy as a treatment for this rare genetic disorder. The page discusses…
According to a story from Hematology Advisor, the drug luspatercept (marketed as Reblozyl) recently demonstrated its ability to reduce transfusion burden in patients with beta thalassemia that depend on regular…
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