By Jodee Redmond from In The Cloud Copy Assemblywoman Nicole Malliotakis (R-East Shore/South Brooklyn) introduced Ava Rose Garnett in New York’s Capitol building in Albany recently. Miss Garnett is a…
By Danielle Bradshaw from In The Cloud Copy Sean and Josie Kelly, a New York native couple, have had a newborn son named Sawyer who was born with a rare…
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…
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…
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…
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 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.…
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…
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…
As noted recently in an article published by Cancer Network, the FDA has cleared CYNK-001 for use in COVID-19 patients. When the investigational new drug (IND) application is submitted to…
According to a story from Live Science, the ongoing coronavirus/COVID-19 pandemic has upended daily life in a myriad of ways and can make leaving the house feel a little intimidating.…
Alex Biagi was planning to try out for his high schools basketball team, and could already dunk. He said his dream was to be a professional basketball player. He grew…
According to an article in the Asco Post, the FDA granted accelerated approval to Merck's KEYTRUDA®, pembrolizumab, for the treatment of metastatic small cell lung cancer (SCLC). The drug targets…
On Wednesday April 22nd, 2020, the World Orphan Drug Congress held a one hour webinar that was focused on how the rare disease community could respond to the ongoing coronavirus/COVID-19…
According to Psychology Today, French physicians may have discovered new early symptoms of COVID-19: dyshydrosis and urticaria. Though the article is published in French, you can read the dermatologists'…
As reported in the CCA Blog, COVID-19 is reminding everyone what self-care means. When we can't rely on turning outwards to seek joy and comfort and good health, we must…
COVID-19 is difficult for many of us. We are facing an unprecedented time and questions abound. How do we handle our work, our families, our health? If you are a…
The ME Association has recently published its latest weekly "research roundup" for the week of April 17th. This includes the latest research related to myalgic encephalomyelitis (ME), also known as…
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