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.
Arcus Biosciences and Gilead Sciences issued a press release announcing encouraging overall response rates and six-month progression-free survival rate for their combination treatment of domvanalimab plus zimberelimab and chemotherapy…
Continue ReadingEncouraging Findings in Phase 2 Gastric Cancer Clinical Trial
Cancer immunotherapy has transformed the landscape of cancer treatment, revealing that not all patients with solid tumors respond effectively to this approach. As reported by News Medical Life Sciences,…
Continue ReadingNanocapsules Enhance Immune Response Against Cancer Tumors
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…
Continue ReadingScientists Demonstrate Gut Microbiome Connection with Transfer of Alzheimer’s to Healthy Animals
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…
Continue ReadingPromising Interim Data in Phase 1/2 Hunter Syndrome Trial
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,…
Continue ReadingWave Life Science’s New Trial Application is Causing Renewed Excitement Around RNA Editing
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…
Continue ReadingInfantile Spasms: Mom Saves Baby’s Life After Being Dismissed by Two Hospitals
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…
Continue ReadingAnalysis: Reasons for Small Cell Lung Cancer Patients Refusing Treatment
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…
Continue ReadingScientists Are Calling Monkey’s Two-Year Survival After a Pig Kidney Transplant “Unprecedented”
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…
Continue ReadingResearchers Seek Reason for a Higher Survival Rate in Obese Patients with Pulmonary Arterial Hypertension (PAH)
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…
Continue ReadingTwo CTCL Trails on Partial Hold After Patient Death
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…
Continue ReadingOver 200 Rare Diseases to be Included in Genomics England Research Study
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…
Continue ReadingFDA Grants Approval for Innovative Dual-Compound Therapy in Pompe Disease
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…
Continue ReadingThe FDA Has Approved the First Blood Test to Identify Multiple Hereditary Cancers
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…
Continue ReadingHeligenics Finds Variants of IFNB1, Used to Treat Multiple Sclerosis
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,…
Continue ReadingDeath of Muscular Dystrophy Patient Potentially Caused by Adeno-Associated Virus That Delivered 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…
Continue ReadingHairy Cell Leukemia: Drug Combo Achieves Significant Remissions
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…
Continue ReadingCervical Cancer is on the Decline Worldwide but on the Rise in India
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…
Continue ReadingIonis Pharmaceuticals Announces Positive Results in Familial Chylomicronemia Syndrome
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…
Continue ReadingWilsons Disease: Researchers Survey Care of Patients in German University Centers
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…
Continue ReadingOsaka University Researchers Study Reasons for Rare Disease Diagnostic Delays
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…
Continue ReadingNewly Approved Treatment Goes Beyond Rivals in Myelofibrosis Anemia
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…
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