As originally reported in the Cambridge Independent; rare disease treatment is full of potential. In part, because there aren’t many. This is not for lack of ability, but for lack…
As originally reported in Medscape; many rare disease patients know the frustration of not having any drugs or treatments available for their disease. 95% of rare disease patients have no…
If you are unable to work because of a medical condition, you may qualify for disability benefits from the Social Security Administration (SSA). The SSA oversees two different disability programs, Social Security Disability…
According to a story from BioPortfolio, the drug companies bluebird bio, Inc. and the Bristol-Myers Squibb Company have recently announced positive final results for their phase 2 clinical trial. This…
According to a story from globenewswire.com, the biopharmaceutical company Lexicon Pharmaceuticals has recently announced that it has completed its safety review of the initial cohort for its phase 2 clinical…
According to a story from mHealth Intelligence, the University of Alabama has found success with using telehealth to check in with kidney disease patients that are on dialysis. Now, it…
A drug created by Agios Pharmaceuticals, TIBSOVO, was recently granted the Breakthrough Therapy designation by the FDA for the treatment of relapsed or refractory myelodysplastic syndrome (MDS) with an…
According to a story from Pulmonary Hypertension News, a recent study has determined that cardiopulmonary exercise testing can be an effective method to help identify systemic sclerosis patients that are…
Happy Holidays, Zebra Community! As we gear up for the holiday season, we're highlighting four articles about managing rare disease during the holidays. We have some new articles about spending…
According to a story from Medscape, an advisory panel from the US Food and Drug Administration (FDA) has recommended the approval of the drug teprotumumab for intravenous administration as a…
Mantle cell lymphoma (MCL) is a very rare form of cancer, and oncologists may only see a few cases of it through the course of their careers. Because of…
According to a story from PR Newswire, the biopharmaceutical company Soligenix, Inc. has announced recently that its phase 3 clinical trial has officially completed enrollment. This trial is testing the…
According to a story from the Star Tribune, being disabled in Minnesota can be a damaging affair. The state's most common method for caring for disabled people are group homes,…
According to a story from prnewswire.com, the biopharmaceutical company InMed Pharmaceuticals, Inc. has recently announced that that company's Clinical Trial Application has been approved in the Netherlands. This approval will…
There is no known cure for sickle cell disease. Treatment is symptomatic and meant to prolong life. While a blood and bone marrow transplant is an option, not everyone…
The holiday season and end of the year/decade is upon us! As schedules slow down and we begin to reflect, I too began to take stock of the experiences that…
According to a story from Medical Xpress, a recent study has confirmed the status of palmoplantar pustolosis as a rare, orphan disease. A recent study of patients with the disease…
According to a story from globenewswire.com, the drug developer Agios Pharmaceuticals, Inc. has recently released a preliminary analysis of phase 2 trial findings that have served to establish proof-of-concept for…
By Caitlin Seida from In The Cloud Copy 2019 was a record year for drug recalls due to safety and quality concerns. It’s also been a record year for the…
By Caitlin Seida from In The Cloud Copy Medical alert dogs can give their companions a newfound sense of independence and freedom. Enter Bill, a golden retriever owned by Laura…
Treatment options for Gorlin syndrome are often surgical, meaning that patients face possible scarring, loss of facial function and structure, and lowered quality of life. PellePharm aims to change…
By Rachel Whetstone from In the Cloud Copy Pete Frates, known for being an Amyotrophic Lateral Sclerosis (ALS) advocate and an inspiration for the Ice Bucket Challenge, has passed away…
.jpg)
Dystrophic epidermolysis bullosa (DEB) is a condition that has many sub-types, one of which is pretibial. The COL7A1 gene, which is responsible for producing VII collagen, is the cause of…
Philippe Pakter’s daughter Lysiane was born on March 29th 2017 in Lyon, France. However, soon after her birth, Philippe and his partner Delphine were hit with shocking news: Lysiane was…
Happy Thursday, Patient Worthy Friends! This week we're highlighting two stories on families who went on a diagnostic odyssey to understand their children's rare diseases. Additionally, we have some big…
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.
