Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.
Michael Myers, M.D., CEO of the Quoin pharmaceutical company describes Netherton Syndrome as a devastating skin disorder that at times may be fatal. In a recent article published in BioSpace,…
Cognitive deficits have been recognized as symptoms of advanced liver disease for many years. As reported in Bioengineer, the American Journal of Pathology recently published a study providing insights…
The 64th ASH Annual Meeting held on December 10, 2022, focused on various approaches for hematologic diseases with the goal of improving quality care and outcomes. Dr. Stephanie Lee, of…
Jenny Decker of Fenton, MO. interviewed with KMOV4 explaining that she was unable to walk when she was three years old due to a rare disease called Charcot-Marie-Tooth disease. Doctors…
Sangamo Therapeutics, with its primary focus on genomic medicine, recently issued a news release announcing data from its phase 1/2 STARR trial. The data evaluated isaralgagene civaparvovec, or (ST-920) as…
Rare disease stakeholders are called on to participate in the first Rare Disease Therapeutic Alliance of 2023 featuring an agenda presented by Rare Advocacy Movement experts. The agenda will…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
423 words 10% matched vs 524 words 5% matched The Rare Diseases International Organization reports that the groundbreaking UN Resolution on Persons Living With a Rare Disease (PLWRD), effective…
One year ago, in December 2021, clinical studies of lovo-cel for patients who were eighteen or younger were put on partial hold by the FDA. At the time, bluebird…
Continued From Part One Multiple myeloma is caused by the buildup of abnormal white blood cells that form tumors found primarily in bones. Available treatments work for short periods…
Globe Newswire recently published an article announcing positive results from Amolyt Pharma’s Phase IIa proof of concept clinical trial of AZP-3601. Amolyt Pharma, Cambridge, Massachusetts, and Lyon, France, is a…
Part 1 Newswise recently published exciting news of a therapy that causes the immune system to destroy cancer cells in the bone marrow. Researchers at Icahn School’s Tisch Cancer Center…
Waldenstrom macroglobulinemia (WM) is defined as a B-cell lymphoplasmacytic lymphoma. Data from patients in Latin America on WM are scarce. Researchers are hoping to fill the void by analyzing…
The latest statistics on heart disease and its effect on Black communities are startling. According to an article in the Dallas Examiner, Black people are fifty to seventy percent more…
A team of researchers at Shanghai’s Fudan University, acting upon the knowledge that ketogenic diets can boost liver ketone production, have recently examined the possibility of preventing thrombocytopenia (low…
Do you wish to be heard? Recently PR Newswire announced that on the 28th of February 2023, NORD and the worldwide community of rare disorders will be raising awareness…
Contrary to simply stopping tumor growth, the biomedical engineers at Duke University have successfully demonstrated a new treatment for pancreatic cancer in a mouse model that may make a…
Med City News published an article by Bruce Feinberg, M.D. at Cardinal Heath, examining CAR-T treatments and the impact they have had in medicine during the past five years. CAR-T's…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
In 2021, Alyssa, who is now thirteen years old, and her family were told that she had acute lymphoblastic leukemia. Alyssa did not respond to either the standard leukemia…
According to a recent article featured in Biospace, researchers found substantial evidence of a biochemical correction after treating an eleven-year girl for Gaucher Disease. This is the first pediatric case…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
Researchers at Brown University, in collaboration with an MIT research team, discovered a method of penetrating the blood-brain barrier (BBB) in mice and delivering medication to treat lethal tumors…
Have you ever wondered how a rare disease can take control of a person’s life? The deteriorating health, being unable to receive an accurate diagnosis, and more importantly finding an…
Genetech recently announced the FDA’s approval of Tecentriq to treat alveolar soft part sarcoma (ASPS) pediatric and adult patients over the age of two. This first-ever treatment has been developed…
© Copyright 2024 Patient Worthy
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.
