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.
.jpg)
According to an article in the Alzheimer’s publication Being Patient, participants who had been enrolled in the discontinued phase three trials of aducanumab will start receiving the drug as…
.jpg)
According to a January 2020 release by Business Wire, the USFDA has awarded accelerated approval of Epizyme’s TAZVERIK™ in the treatment of advanced epithelioid sarcoma (ES). The approval was…
.jpg)
Janssen R&D, a division of Johnson & Johnson (J&J), has partnered with the UK Biobank and others according to a recent news release by 3BL Media. It is noteworthy…
.jpg)
Although ‘precision medicine’ has been mentioned in medical circles since 1999, according to a recent article in Pharmafile, technological advancements have now made it feasible. For years physicians have…
.png)
Researchers at the Medical Center of Hackensack University conducted a study of three drugs now proven to be safe and effective for the treatment of severe seizures. According to…
.jpg)
According to a recent article in the MalayMail, it occurred to Abdullah’s doctor that the 67-year-old would be an inspiration for others who have kidney failure. His nephrologist, Dr.…
.jpg)
According to a recent article in MedPage Today’s 2019 year-end review, the number of life-threatening lung diseases in systemic juvenile idiopathic arthritis (sJIA) patients has been increasing. sJIA is…
.jpg)
Genentech (Roche Group) announced positive results of its FIREFISH trial in a recent Business Wire news release. The second part of the study evaluated risdiplam’s efficacy on infants ages one…
It has been evident to scientists for years that Alzheimer’s disease, which affects about five million people in the U.S., is caused by an accumulation of two proteins within…
.jpg)
According to a recent article in the UK Newcastle Chronicle, when doctors gave Leanne Buckley an opportunity to terminate both her pregnancies, Leanne and her husband Michael were emphatic…
Surgery has been found to often be the best option towards a cure for a cancerous tumor (resectable) but there are many other tumors that are diagnosed as unresectable…
.jpg)
According to a recent article in PsychCentral, a rare genetic disorder called 22q11.2 deletion syndrome (22q) is very often misdiagnosed. A study shows that social impairment is the most…
.jpg)
The biopharmaceutical company Cellectar Biosciences recently announced that the FDA granted its lead phospholipid drug conjugate, CLR 131, Orphan Drug Designation(ODD) for treatment of lymphoplasmacytic lymphoma. ODD is granted by…
.png)
The American Journal of Managed Care (AJMC) published excerpts from a presentation by Dr. Andrew Wei at the 61st American Society of Hematology (ASH) Annual Meeting. The article stated…
.jpg)
According to a recent article in Pharma Tutor, researchers at NHGRI, a division of the National Institute of Health, identified the biological cause of a newly-discovered autoinflammatory disease they named…
.jpg)
According to a recent article in Yahoo News, Johan’s mother’s seven-year-old brother died years ago of chronic granulomatous disease (CGD). The mother, Maren, was told that she also had…
.jpg)
Ty Eschenbaum’s world of high school football and working on his family’s ranch and farm changed suddenly after his freshman year when he was diagnosed with leukemia. Ty, who…
According to an article in BioSpace, the FDA recently approved the PARP inhibitor Lynparza that was developed by Merck and AstraZeneca to be used as maintenance therapy for pancreatic…
.jpg)
Susan Winn’s family history of ovarian and breast cancer can be traced back to the 1860s. Susan’s adult daughters, Kathryn, Bridget and Maureen, were recently interviewed with CURE® (Cancer Updates Research…
.jpg)
An article published earlier this year by Fierce Biotech outlines an approach that was developed by researchers at the Fred Hutchinson Cancer Research Center. Using CRISPR-Cas9’s gene-editing method the researchers were…
.jpg)
According to a recent article in Euronews, this year one hundred children who were diagnosed with spinal muscular atrophy (SMA) will receive Zolgensma, the world’s highest-priced drug, at no cost.…
.jpg)
According to recent articles in Runner’s World Magazine, having a diagnosis of multiple sclerosis has not stopped Cheryl Hile, Jennifer Lee or Melissa Ossanna. Each woman is fighting the symptoms…
.jpg)
According to the UK’s National Institute for Health Research (NIHR), a new technology called machine perfusion (MP) has the potential to solve organ shortage, a major challenge in liver transplantation.…
An open letter to The Editor from Gaetani et al was recently published in the Orphanet Journal of Rare Diseases. Dr. Gaetani and associates at the Fondazione Policlinico Universitario, Rome Italy,…
.jpg)
Pfizer issued a statement this week through its Media Relations that its drug VYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy received positive opinion by the European Medicines Agency’s (EMA) Committee for…
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.
