For many years, scientists have known about toxic clumps forming in patients with Huntington’s disease (HD). However, until recently, scientists relied on examining post mortem brain samples of HD…
January is not a surprising month. It takes people a while to remember to write the correct year, your Facebook feed is full of people going hard at the gym,…
The Phase 3 BE MOBILE 2 clinical trial sought to evaluate the safety, efficacy, and tolerability of bimekizumab for adult patients with ankylosing spondylitis (AS), an inflammatory disease affecting…
Have you ever wanted to just take a break from addressing your illness? Like, taking those medical binders and folders and just keeping them in your car trunk for a…
In a December 13 press release listed on the company's website, genome editing company Intellia Therapeutics, Inc. ("Intellia") shared that the first patient had been dosed in a Phase…
According to Dr. Deepak Chellapandian, a Johns Hopkins specialist in bone marrow transplant for rare diseases, the prognosis for his patient was very poor. Jocelyn was born with chronic…
According to DocWireNews, on December 16, 2021, the U.S. Food and Drug Administration (FDA) granted Accelerated Approval to Tarpeyo (budesonide) delayed-release capsules. The therapy is designed to treat adult…
Washington University Researchers readily acknowledge the attributes of sonodynamic therapy (SDT) as a promising therapeutic method to treat hepatocellular carcinoma (HCC). SDT is noninvasive with high tissue penetration, but…
Dr. Richard Stone recently interviewed with Targeted Oncology to discuss challenging acute myeloid leukemia subtypes and a list of available therapies. Dr. Stone was asked to look back at…
Clinical trials can be extremely beneficial in learning more about various diseases, as well as the safety, efficacy, and tolerability of potential treatments. According to a news release from…
Pancreatic cancer so far has been able to escape early detection. When it is discovered, it is usually in an advanced stage and difficult to treat. According to a…
On December 17, 2021, global immunology company argenx SE ("argenx") shared on its website that its therapy VYVGART (efgartigimod alfa-fcab) was approved by the FDA for the treatment of…
Continued from Part One With time my speech improved and eventually I could speak fluently again. I also worked with an occupational therapist who helped teach me how to…
Discovering a treatment for a rare disease is challenging and rewarding, especially when it is an extremely rare disease such as perivascular epithelioid cell tumor (PEComa). Biospace recently carried…
In a news release from mid-December 2021, rare liver disease company Albireo Pharma, Inc. ("Albireo") shared that positive topline data was available from a Phase 1 clinical trial evaluating A3907…
According to a December 16, 2021 news release from biopharmaceutical company Applied Therapeutics, Inc., the company has initiated a registrational Phase 2/3 INSPIRE clinical trial to evaluate AT-007 for patients…
Thinking about the year-end holidays brings up a kaleidoscope of images, and an array of emotions for me. And everyone else too, I imagine. There are the vivid memories of…
According to a story from globenewswire.com, the gene therapy company Sio Gene Therapies, Inc., has recently released encouraging interim findings from its phase 1/2 clinical trial. This trial is evaluating…
Gareth Crabb is a thoughtful and caring six-year-old boy from Swansea, U.K. He is spending his second Christmas in the hospital this year, as he has anaplastic large cell…
Although most people are familiar with the term ‘hepatitis,’ many are not aware that it is an autoimmune disease. A recent article in Life Sciences describes autoimmune hepatitis as a…
In May 2016, I was diagnosed with a rare genetic neurological disease called adrenoleukodystrophy (ALD). Sadly, it's incurable and there's no medication to ease my condition. Since then, my life…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. These are defined as conditions affecting under 200,000 Americans.…
By Rachel Whetstone from In The Cloud Copy Food is fundamentally linked with celebrations in every culture around the world. Families gather for a feast at big holidays and birthdays…
According to a news release from RNA therapeutics company ProQR Therapeutics N.V. ("ProQR"), the first patients have been dosed in two Phase 2/3 clinical trials evaluating QR-421a for USH2a-mediated retinitis…
For those of us who suffer from depression, that mostly invisible mental illness that, at its worst, can render us incapable of getting out of bed and, at its best,…
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.
