According to a press release from Verrica Pharmaceuticals and its partner Lytix Pharmaceutical via Healio, results of Part 1 of the phase 2 study of their investigational therapy candidate VP…
A 15-year search for a diagnosis by Lilly Grossman and her parents ended in 2013. Lilly became the first person in the world to be diagnosed with ADCY5-related dyskinesia.…
Currently available therapeutics may not be adequately effective in the fight to treat late-stage acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). Researchers and drug developers are working to identify…
According to a story from Axios, the relatives of Henrietta Lacks are filing a second lawsuit against a biopharmaceutical company, alleging that the company is unfairly profiting through the use…
Galila Yohannes’ parents, who originally hail from Eritrea, brought their family to Israel for a better life. And while they never would have expected what came next, this move likely…
According to a story from Medpage Today, a study conducted by scientists at the University of Texas MD Anderson Cancer Center highlighted the substantial benefits of aerobic exercise in people…
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…
An Intern’s Journey When my summer internship with Red Nucleus began, I had little familiarity with rare diseases and the life science industry. A distant relative of mine struggled with…
The U.S. FDA has approved two treatments for people living with idiopathic pulmonary fibrosis (IPF). These therapies—Esbriet (pirfenidone) and Ofev (nintedanib)—both work to reduce fibrosis, or scarring, in the…
In Jordyn Sava's reporting from Targeted Oncology, readers learn that the U.S. Food and Drug Administration (FDA) granted clearance to a global Phase 3 study. This means that the…
Unfortunately, gaining access to care within the rare disease space can be difficult. There is often lesser education and awareness about rare conditions, less research performed, and poorer access to…
21-year-old Chloe Feighery has a deep love and appreciation for her younger sister Heidi, who is just one and a half years old. When she learned that she was becoming…
Soon after Emma Young was born, she was diagnosed with a rare genetic disorder called Bannayan-Riley-Ruvalcaba syndrome (BRRS). At two months old, she battled COVID-19. Then she was diagnosed with…
For the first year of his life, Warner Kays looked like an average, healthy, happy baby. He crawled around, took joy in family time with his older sister Presley, and…
Have you ever heard of CD55-deficient protein-losing enteropathy? This rare condition, which is also called CHAPLE disease, was only identified in the past 10 years. Right now, there are only…
A recent press release from Verrica Pharmaceuticals and its partner Lytix Biopharma reported in Healio announces the results of the first portion of a study evaluating VP-315 to treat basal…
When Crystal York learned that her daughter Harmony had a rare genetic disease called DHX30, she was shocked. Even more shocking is that Harmony is just one of 40…
The life of the four-year-old was saved. The operation conducted at Medcare Orthopedics was a success. It was the first of its kind to be performed in the UAE.…
Enterome presented its findings to the 17th International Conference on Malignant Lymphoma (ICML) in June of this year. As reported in the GlobeNewswire, the company demonstrated that E02463 together…
The later part of 2023 will see decisions on drugs in four areas such as advanced melanoma, sickle cell disease, Alzheimer’s disease, and depression. BioSpace shines the spotlight on…
Chorea, a characteristic manifestation of Huntington's disease, refers to uncontrolled "dance-like" movements in the fingers, feet, face, or torso. These movements may worsen in times of heightened emotion or…
At the moment, there are limited treatment options for glioblastoma, a rare form of brain cancer. While surgery, radiation, chemotherapy, and electric-field therapy are all utilized in the fight…
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…
The Phase 2 EXHALE-1 clinical trial, initiated by clinical-stage biotechnology company Areteia Therapeutics, Inc. ("Areteia"), sought to understand how safe, effective, and well-tolerated dexpramipexole was in reducing symptoms of…
My name is Nicholas Alves, and I am a 26-year-old male from Massachusetts. I have a completely life destroying condition called PSSD, or post SSRI sexual dysfunction. PSSD can arise…
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