Happy 2nd Birthday to TAPS Support!
Patient Worthy would like to wish a Happy 2nd Birthday to one of our partners, the TAPS Support Foundation. It is hard to believe that it has only been two…
Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
Patient Worthy would like to wish a Happy 2nd Birthday to one of our partners, the TAPS Support Foundation. It is hard to believe that it has only been two…
Don't forget to read Part 1 of our interview, where we discussed what Costello syndrome is, Ryan's son Reynolds' diagnosis, and what spurred the family to want to make a change. mejo…
In 2020, researchers estimated that 627,000 people died of malaria. Unfortunately, many of these deaths were children – particularly in areas such as sub-Saharan Africa. Scientists and researchers have been…
Before you read on, make sure you take a look at Pt. 1 of our interview, which goes over what PGAP3 is, Lucy's story, and the reasons behind why Zach and Geri…
Over the last few years, the incidence of nonalcoholic fatty liver disease (NAFLD) has increased rapidly. As a result, many researchers are beginning to pay closer attention to this disease…
“My son has Costello syndrome, but he is not defined by his diagnosis,” Ryan Sheedy tells me about his son, Reynolds. To Ryan, his statement exemplifies his beliefs about needed…
The incidence of nonalcoholic fatty liver disease (NAFLD) has been rapidly rising worldwide, with many healthcare providers expressing concern. Because of this rapid growth – and simultaneous problems with underdiagnosis…
Zach and Geri Landman are devoted parents and physicians – a pain specialist and pediatrician, respectively – committed to making the world a better place for those around them. They…
Currently, there are upwards of six million people in the United States alone living with Alzheimer’s disease. As some researchers work to determine the exact cause of this neurodegenerative…
Have you ever heard of machine learning? Machine learning is a form of artificial intelligence (AI) which teaches systems to learn, act, and improve an experience without explicit programming.…
Could a vaccine help treat cancer such as Merkel cell carcinoma (MCC)? Immunomic Therapeutics believes so. Its therapeutic plasmid DNA (pDNA) vaccine, ITI-3000, is designed for those with MCC,…
Unfortunately, doctors and scientists are still learning about Zellweger syndrome, a rare multisystem genetic disorder. But this can be incredibly difficult for families whose children are diagnosed – after all,…
Desiree Thompson recently turned three years old – and the Tulsa County Sheriff’s Office (TSCO) planned a big surprise for her. You see, Desiree, who has epilepsy and lissencephaly, absolutely…
As many people in the rare, chronic, or underserved disease community know, the journey to diagnosis can be a long and difficult one. For Texas resident Cheryl Yetz, the diagnostic…
Currently, there are an estimated 30 million people in the European Union (EU) who are living with a rare disease. Drug developers are incentivized to create therapies for those…
Currently, clinical-stage oncology company Sumitomo Pharma Oncology, Inc. is working to develop DSP-5336 for patients with acute myeloid leukemia (AML). The company even began recruiting for a Phase 1/2 study…
In an August 3rd, 2022 news release from Syros Pharmaceuticals (“Syros”), the company shared that its product tamibarotene had received a positive opinion for its Orphan Drug designation application.…
In a news release from biopharmaceutical and RNAi therapeutics company Alnylam Pharmaceuticals, Inc. ("Alnylam"), the company shared that positive data was available from the Phase 3 APOLLO-B clinical trial.…
For most of his life, Jack O’Donnell was as healthy as can be. Even as he headed towards Oklahoma State University, ready to study engineering graphics and design drafting, Jack…
In the U.S., the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. “Rare” refers to any condition affecting fewer…
Kris and Carla Thompson were so thrilled when they learned that they would be expecting a daughter. When Lucy was first born, Carla describes her as a cheeky, upbeat, and…
According to a news release from biotechnology company Kira Pharmaceuticals, its therapy KP104 earned Orphan Drug designation in the United States. KP104 is in development as a therapeutic option…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare or life-threatening conditions. A “rare” condition is defined as…
An article published by Ophthalmology Times Europe shared that the first patient had been dosed in the Phase 2 STARLIGHT clinical trial evaluating MCO-010 as a potential therapeutic option…
When Mercedes Young found out that she was pregnant with her son Milo, she knew that she would do everything possible to protect and support her son. Milo was born…