Phase III of the CheckMate -9ER trial has reached its primary endpoint of progression free survival in those with advanced or metastatic renal cell carcinoma. This study was evaluating CABOMETYX…
by Danielle Bradshaw from In The Cloud Copy Hereditary angioedema (HAE) is a rare condition in which a person has swelling beneath their skin (which can occur in any part…
According to Angioedema News, the European Medicines Agency (EMA) is now reviewing berotralstat as a potential therapy to combat severe inflammation attacks in patients with hereditary angioedema. Hereditary Angioedema Hereditary…
A new resource guide has been developed for X-linked hypophosphatemia (XLH) by Prime, a medical education firm. This project has been supported by Ultragenyx Pharmaceutical Inc. The guide provides resources…
The World Health Organization and its partners recently launched the Solidarity international clinical trial in search of an effective treatment for COVID-19. The rising death toll for a virus that…
The month of May is Huntington's Disease Awareness Month, in which a special effort is made to improve awareness about this devastating rare disease. This year, the Huntington's Disease Society…
By: Natalie Homan from In The Cloud Copy Connie Elson was a happy, independent little girl before she started showing worrying symptoms at the age of four. After many doctor…
By Jodee Redmond from In The Cloud Copy Hannah Witton, a YouTuber with 600,000 subscribers to her sex and relationships channel, got tired of hearing comments that people with disabilities…
According to a story from Buzzfeed News, the ongoing coronavirus/COVID-19 pandemic has put the brakes on critical rare disease research all over the globe, ranging from early, highly experimental studies…
The FDA recently granted Orphan Drug status to CS1, a drug therapy from Cereno Scientific, according to Pulmonary Hypertension News. CS1 is designed to provide safe and effective treatment for…
A recent study conducted by a government agency, NIAID, offered preliminary evidence of Remdesivir’s benefit to COVID-19 patients. The drug is heralded as the first antiviral drug to improve COVID-19…
Maddie Nordhoy has a smile that can light up the room. She constantly brings joy to those around her, especially her family. In fact, she is even the inspiration for…
According to a story from the CureDuchenne Blog, CureDuchenne founder and CEO Debra Miller announced that the organization has recently entered into a new partnership with the biotechnology company Myosana…
The Connect MPS Patient Registry was created in 2016 to learn more about Sanfilippo syndrome and mucolipidoses (ML). 24 advocacy organizations are a part of this registry, all of which…
HAE is not my story. But it’s definitely part of my story. Looking back, I realize that my late childhood and early teen years held some occasional, unexplained swelling events that…
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 .…
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