The Foundation for Angelman Syndrome Therapeutics (FAST) recently entered a partnership with Rush University in order to establish a clinical trial and translational research center for rare neurodevelopmental disorders.…
Make sure to read Part 1 of Kandise's story. In Part 1, she discusses some of the symptoms of hereditary multiple exostoses (HME), as well as how she finally reached a diagnosis.…
Contributed by Anna Ellis Every February 28, millions of people around the world participate in Rare Disease Day to raise awareness about the more than 10,000 identified rare diseases that affect…
At nearly 33 years old, Kyla McGaughey has overcome more challenges that many people can imagine. Her medical journey began in 2019 and it took over two years for her…
When Richard Poulin's daughter Rylae-Ann was born, it wasn't long before he and his wife began to wonder if something was wrong. They both felt confident in understanding the…
Ready to read? Before you do, make sure to check out Parts 1 and 2 of Tracy's story. In Part 1, Tracy describes the symptoms that led her to pursue a diagnosis. Part 2…
Clinical trials can play a crucial role in understanding diseases, as well as identifying potential therapeutic options. This is especially important in newly identified or poorly understood conditions such as…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Prior studies have suggested that metformin, often used as a first-line treatment for people with type 2 diabetes (T2D0, could confer benefits for Rett syndrome. In particular, prior studies found…
Summer Wroniecki fell in love with dancing at a young age. At just 2.5 years old, she was already taking over the dance floor at Highland Dancing; her teachers were…
In the past, research has found connections between polycystic ovary syndrome (PCOS) and pancreatic cancer. For example, one study suggested that having PCOS could increase pancreatic cancer risk by…
Before you read on, make sure to check out Part 1 of Tracy's story. In Part 1, Tracy discusses the symptoms that led her to pursue a diagnosis and the multi-year diagnostic…
Lauren Bruccoleri and her husband Matthew weren’t initially planning on having their son Grayson. But when Lauren found out that she was pregnant, she was overjoyed. However, Lauren and Matthew…
When Kandise MacLeod was twelve years old, she began noticing various growths and tumors popping up on her bones. These sometimes caused pain or discomfort; in one case, Kandise even…
Before you read on, make sure to check out Part 1 of our interview with Christina ("Chris") Coates. In Part 1, we discussed Chris' Cavernous Malformation, surgery, and diagnostic journey to hypertrophic…
Seizures associated with conditions such as Dravet syndrome and Lennox-Gastaut syndrome (LGS) can be difficult to treat; these seizures may be treatment-averse and may not respond well to current anti-epileptic…
Have you heard of the pulmonary microbiota, which may also be referred to as the lung microbiome? This refers to the complex collection of microorganisms (bacteria, viruses, bacteriophages, fungi) that…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In 2018, Libby Porter welcomed her first child into the world. Her family was thrilled, especially her father John, who was so excited to have a granddaughter. But things…
Dr. Seth Lederman founded Tonix Pharmaceuticals with a deep desire to change the treatment landscape for patients. The company’s rich pipeline features therapeutic options for fibromyalgia, post-traumatic stress disorder (PTSD),…
Christina (“Chris”) Coates has lived a full, joyous, and fulfilling life. The Arizona native has built something beautiful: a family with two daughters, two cats, two dogs, and a loving…
The advocacy group Just Treatment recently published a press release titled 'BREAKING: Cystic Fibrosis Patients Launch Global Challenge to Vertex Monopoly on CF Drugs.' Vertex Pharmaceuticals has taken the lead…
This week the family of actor Bruce Willis announced that he received a diagnosis of frontotemporal dementia (FTD), a rare disease that causes behavioral changes, language, speech, and memory…
Ten years ago, Tracy Kyttle moved from New England to the DC Metro Area to pursue a career in education leadership. She loved the area’s duality: its proximity to nature…
Before you read on, make sure to check out Part 1 of Laura's story. In Part 1, Laura shares the diagnostic journey, why her family is so rare, and what Carpenter syndrome…
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