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…
According to a news release from Biospace, recent data from the KALM-2 Phase III trial highlighted how Korsuva, an injectable, is beneficial in treating severe itching associated with severe chronic…
Armené and Michael Kapamajian live in LA. Their son Sasoun was diagnosed with severe combined immunodeficiency (SCID). This means that his immune system is compromised and he is extremely vulnerable…
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…
Trevor O'Sullivan Trevor O'Sullivan, who lives in Ireland, has cystic fibrosis (CF). He has undergone two liver transplants. He was the 42nd person in the country to have this surgery.…
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…
Mark Cohen is a 28 year old lawyer who has a rare eye disease called choroideremia. Its genetic, and his grandpa, brother, and cousin were all diagnosed. He didn't receive…
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…
Social distancing sucks for everyone. Even introverts (like myself) are becoming antsy. Similarly, the coronavirus is scary for everyone, even those less likely to be effected by serious illness. However,…
By Jodee Redmond from In The Cloud Copy At one time, hearing a cystic fibrosis (CF) diagnosis was tantamount to a death sentence for a young person. The median survival…
When the 31-year-old woman showed up to Massachusetts General Hospital with a nasty respiratory infection, she was somewhat used to it. In fact, she had been plagued with respiratory problems…
Because I will be looking for a job from a rare but certainly not unique place of being, I decided to reach out to a mentor who lives with the…
U.S. company, Vertex Pharmaceuticals, is the creator of three treatments for cystic fibrosis: Orkambi, Symdeco, und Kalydeco. These drugs were unavailable in Switzerland, as Vertex and Swiss health officials were…
Recently, says Medical XPress, an international research team discovered that the drug therapy 2-deoxy D-glucose can slow cell growth. In patients with ovarian cancer, the drug can halt the growth…
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…
Según un artículo en The Asahi Shimbun, científicos japoneses han desarrollado una nueva vacuna para mitigar la degeneración neuronal causada por la enfermedad de Alzheimer. La vacuna, administrada por vía…
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…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
