In a late May 2023 news release from clinical-stage biotechnology company Acepodia, the company shared that the first patient has been dosed in a Phase 1 study. Within this…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
Managing dystrophic epidermolysis bullosa (DEB) can be difficult. DEB is a rare genetic condition which causes fragile, easily blistered skin. There are multiple DEB subtypes with their own inheritance…
An announcement from the drug company Genentech highlights the results of a recent study which demonstrated that the drug faricimab-svoa (marketed at Vabysmo) was able to dry retinal fluid more…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Ventilatory assistance, NG tubes, tracheostomy: all of these may be used to care for infants born with bronchopulmonary dysplasia (BPD), a chronic lung disease that most commonly affects preterm…
Unfortunately, there are a number of negative or false societal narratives around the concept of “rare disease” or “disability,” particularly when it comes to what these individuals can achieve. Having…
In December 2022, Liam Hendriks—a pitcher for the Chicago White Sox—was diagnosed with non-Hodgkin’s lymphoma (NHL); he shared this news with the world in January 2023, which Patient Worthy reported…
As reported in Yahoo! Finance, the United States’ Food and Drug Administration (FDA) recently granted Orphan Drug designation to MP1032. This therapy, developed by clinical-stage biotech company MetrioPharm, is being…
At first, the round black dots that Carmen saw spattered across the bathroom sink were frustrating, but not concerning. Since her grandchildren were staying with her, Carmen assumed that something…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
The AACR Annual Meeting 2023 took place from April 14-19. During the meeting, stakeholders from across the cancer community—from patients to clinicians—come together to discuss trends, research, and clinical practice.…
The biotechnology company Anixa Biosciences, Inc. together with the Moffitt Cancer Center, issued a press release on May 22nd stating that the second patient had begun treatment as part…
Thirty-year-old Brendan O’Reilly was told that his rare disease affects only one person in 250,000. He was also told that he is lucky to be alive. Brendan’s symptoms confused…
The Chow family was told that their 33-year-old daughter, Brittney, would have to wait about twelve years for a kidney transplant. There were currently no kidneys available for transplantation. Elizabeth…
What do clinical evaluations, blood tests, urine tests, kidney tissue tests, and imaging tests all have in common? These are all diagnostic measures that doctors may use to diagnose chronic…
The month of May is recognized as Prader-Willi Syndrome (PWS) Awareness Month, and May 27th is International PWS Day. This is a time to spread awareness about this rare disease…
In March the FDA’s expert advisers voted 11 to 2 in favor of approving Roche’s antibody drug Polivy to treat lymphoma patients. According to an article in BiopharmaDive, the decision…
Bariatric surgery refers to surgical procedures that can help people with obesity to lose weight when other means have not worked. This type of surgery can help people to live…
The safety, pharmacokinetics (absorption), and tolerability of the investigational eIF2B activator ABBV-CLS-7262 is being evaluated in a Phase 1b clinical study to treat patients with Vanishing White Matter (VWM) Disease.…
PhRMA Public Affairs recently carried the good news that Governor Holcomb of Indiana has joined the governors of Arkansas and West Virginia by signing a law aptly named 'Share the…
This week PR Newswire carried an announcement by the biotechnology company Immunic Inc. of preclinical data confirming that Vidofludimus Calcium (IMU 838) may be a Nurr 1-Activator reinforcement. Immunic is…
7-year-old Peter Heath loves to tell jokes, play with Legos, hang out with his friends, and spend time outside. Guided by his white cane, which Peter aptly named Marshmallow, Peter…
Last year, 35-year-old Jenna Neduchal ran her first Flying Pig Marathon. This annual event serves to unite the community, create an accessible event for all, and raise funds for important…
There are no cures or treatments available for people with hereditary spastic paraplegia (HSP) type 56, or SPG56. While the current standards-of-care focus on symptom relief and mobility assistance, patients…
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