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.
Scientists have previously suggested that the gut microbiome plays a role in causing Alzheimer’s disease, and this has now been confirmed. A recent report from ScienceAlert covered a study…
Professionals from all corners of the world convene at SSIEM each year to discuss the latest groundbreaking discoveries. According to a report in BioSpace, one such breakthrough was presented by…
If Wave Life Sciences’ recently submitted trial application for WVE-006 is approved, it will become the first RNA editing treatment in a clinical development setting. Wave’s CEO, Paul Bolno,…
Imagine the frustration of Faustina Cavero, unable to follow her maternal instincts that told her something was wrong with her baby. Doctors at two different hospitals disagreed with her and…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
The xenotransplant involved transplanting Yucatan miniature pig kidneys into macaque monkeys with 69 genetic modifications. One monkey survived for over two years, offering hope to the team of Harvard…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
The two trials evaluating lacutamab, an investigational therapy developed to treat T-cell lymphomas, were put on partial hold by the FDA. The patient died of hemophagocytic lymphohistiocytosis, which is…
One of the most successful healthcare services benefiting the public is newborn screening. Unfortunately, this life-saving health initiative is not provided for all rare diseases in some U.S. states. As…
Genomics is revolutionizing healthcare processes, offering the potential to enhance the lives of numerous individuals by enabling the early detection of treatable disorders and providing lifesaving therapies. Every year, thousands…
After years of seeking approval for the treatment of Pompe disease, this week, as reported by GlobeNewswire, the FDA granted approval to its developer, Amicus Therapeutics, for the first…
This week, the USFDA granted a new marketing authorization for the ICHC Cancers Panel, thereby creating a new regulatory classification. According to a report in Inside Precision Medicine, the…
Interferon beta was the first available therapy for treating multiple sclerosis (MS) and is still the most commonly prescribed treatment. This therapy helps reduce relapses in MS patients. Distinguished UCLA…
For over four decades, the aspiration of gene therapy has been to develop novel therapies with the potential to enhance human health. Recombinant AAV (rAAV), a type of gene therapy,…
Early in the 1990s, anti-CD20 antibody therapy marked a significant improvement in the outcomes for patients with B-cell cancers. B-cell lymphoma is a type of cancer that originates in…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
This week Ionis Pharmaceuticals, Inc. issued a press release via PRNewswire announcing topline Phase 3 results for its Balance study of olezarsen. The study ( NCT04568434 ) enrolled people with…
In healthy individuals, copper is absorbed from food, and the excess is excreted by a substance called bile which is produced in the liver. It should be noted that…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
As reported in MedCity News, Ojjaara was part of GSK’s $1.9 billion acquisition of Sierra Oncology last year. FDA approval of GSK’s momelotinib covers the treatment of adult myelofibrosis…
You are invited to join hundreds of thousands of people touched by spina bifida, the most common congenital birth defect causing disability. There is no cure. This is a…
The 2022 Medscape Physician Suicide Report shows that physicians, compared to the general public, have double the rate of suicides. Amna Shabbir, a doctor of internal medicine, felt compelled…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Indie songwriter and singer Sufjan Stevens recently posted on, Tumblr, his favorite media platform, that he has been diagnosed with Guillain-Barré syndrome (GBS). Sufjan is now in the hospital…
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