When it comes to rare diseases, the diagnostic process can be long and arduous. On average, patients wait around 7 years for a diagnosis. Oftentimes, this also complicates treatment and…
One-year-old Alexander was diagnosed with Barth syndrome before he was born; his parents knew to test for the condition after their son Elias passed away from complications in 2018. While…
In a recent news release, biotherapeutics company CSL Behring shared that the company received FDA approval to update the Hizentra label for patients with chronic inflammatory demyelinating polyneuropathy (CIDP). Within…
On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare…
Recently, the European Commission (EC) approved a marketing authorization for ADV7103 (Sibnayal), the first approved treatment for patients with distal renal tubular acidosis (dRTA). In a news release, pharmaceutical company…
The month of May is being recognized as Lyme Disease Awareness Month, a time to spread awareness about this disease among the medical field and the general public. As spring…
No one can truly prepare for life raising a child who has a rare disease. But as exhausting as it can be, Lori Dunham describes that raising her little girl…
In this digital age, rare disease issues have become more prevalent and more visible. According to Eurordis, a rare disease affects less than 1 in 2000 people. There are more…
In a news release from April 2021, biopharmaceutical company Jazz Pharmaceuticals, Inc. ("Jazz") shared that the FDA accepted its supplemental New Drug Application (sNDA) for review in relation to Xywav,…
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.
