According to BioSpace, recent research has demonstrated that CRISPR gene editing could be the answer to treating progeria, or Hutchinson-Gilford progeria syndrome. The Broad Institute and NIH have collaborated to…
Hayden Grosvenor is used to spending time in the hospital. He has Langerhans cell histiocytosis (LCH), a rare condition that is characterized by an excess of white blood cells. Because…
Grant Bonebrake was diagnosed with Alport syndrome at age 12. Since then, he has gone on to advocate for not only Alport syndrome patients, but the rare disease community as…
Monica and Josh Poynter have recently adopted a nine-year-old boy, Trey, from China. They felt a connection to the boy as he has hemophilia type A, the same rare bleeding…
The FDA has recently cleared an Investigational New Drug (IND) application for FBX-101, allowing it to move into a Phase 1/2 clinical trial. This gene therapy was developed for the…
Many people have questions about the COVID-19 vaccine, a number of them pertaining to the speed at which it was developed and approved. Rare disease patients are no exceptions. Barbra…
12-year-old Aidan Carter has Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), a rare, progressive condition that impacts mental development, appearance, physical abilities, and organ function. In order…
Cancer screenings are very important for early detection, which can then lead to treatment administered earlier in disease progression and better outcomes. Pancreatic cancer is no exception. In fact, this…
The COVID-19 pandemic has been difficult for everyone; we've all had to make changes to our lives because of it. Some of us have had to make bigger changes than…
The Neurological Alliance has released a report that details the experiences that rare, neurological disease patients in the UK share and calls for improvements for these people. Experts in the…
Landos Biopharma has recently released data from their Phase 2 trial of BT-11, a treatment for ulcerative colitis. The results were positive, making this therapy a possible viable treatment for…
A study published in BMC Pulmonary Medicine focuses on the role of endothelial progenitor cells and fibrocytes in lung sarcoidosis. They specifically investigated how these cells, along with CD34 + cells, spurred…
According to GlobeNewswire, Health Canada has approved of a biosimilar, Hyrimoz, to treat ankylosing spondylitis and eight other rare conditions. Sandoz Canada is excited about this approval, as it expands…
The FDA has recently approved of GlaxoSmithKline's (GSK) treatment for lupus nephritis (LN), BENLYSTA. This therapy is the first of its kind, meant to treat systemic lupus and adults with…
Albireo has recently initiated their Phase 3 ASSERT trial, according to GlobeNewswire. This study will evaluate odevixibat as a treatment for Alagille syndrome. This therapy fits well with Albireo's mission,…
Before 1983, there were few treatments for rare diseases. Various obstacles stood in the way, including little representation for patients, few incentives, and a lack of resources. Fortunately, the Orphan…
Yash Cary knows what it's like to spend the holidays in the hospital. In fact, three of his last six Christmases have been at UNC Hospital. This is because Yash…
"Once Upon a Christmas Miracle" is a Hallmark movie, falling into their "Movies and Mysteries" holiday series. It tells the true story of Heather Krueger and Chris Dempsey, a couple…
According to an article from Cancer Therapy Advisor, medical professionals have created a new, simplified prognostic score for patients affected by myelodysplastic syndromes (MDS). This score utilizes multiparametric flow cytometry…
Selma Santos has met many nurses over the course of her life. Having a daughter with cystic fibrosis means spending a lot of time in the hospital or at doctor's…
Grief is something that is difficult to go through alone, and this year has brought too much of it. Maya McNeary is no stranger to grief, as she recently lost…
Visual hallucinations have been a known symptom of Parkinson's disease for a long time, but the cause has evaded medical professionals. That is, until now. A recent study, titled "Visual…
Suzanna DePaolis views her life with a rare disease as a battle, one that she is conquering. This battle began fourteen years ago when she was diagnosed with eosinophilic granulomatosis…
Many medical professionals have applauded artificial intelligence and machine learning in the drug development process for a long time, as they can increase the speed while decreasing the cost. However,…
Alina Citovic was diagnosed with spinal muscular atrophy at age five, just three years ago. At the time of her diagnosis, Spinraza was already approved by the FDA. While the…
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