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.
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Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side…
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Vamorolone Has Been FDA Approved to Treat Duchenne Muscular Dystrophy
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In December 2019, three months after his retirement, Paul Goodfellow received a diagnosis of invasive cancer of the bladder. He received three cycles of chemotherapy and then major surgery that…
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Combo Treatment in Clinical Trial Doubles Metastatic Bladder Cancer Survival
In the year 2000, the number of Dengue cases was reported to be approximately one-half million. In October 2023 the total reached 4.2 million cases of Dengue virus. The virus…
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As Temperatures Rise, Dengue Fever is Spreading. Can it be Stopped?
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Every three to four minutes, someone in the U.S. is diagnosed with a blood disease such as lymphoma, leukemia, or sickle cell anemia. As reported in the Pittsburgh Post-Gazette,…
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Mom Signed Up Thousands of Stem Cell Donors in Support of Son with IPEX
The words ‘vigorous exercise’ usually bring to mind running marathons or playing pickleball. Inside Precision Medicine reports that researchers at a Sydney, Australia University tracked the daily activity of more…
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Study Shows an Average of 4 1/2 Minutes of Vigorous Exercise May Reduce the Risk of Cancer by 32%
Chip Wilson, billionaire founder of Lululemon Athletica, was first diagnosed with muscular dystrophy in 1987 at the age of 32. Wilson, who is now 66 years old told Global…
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Wealthy Facioscapulohumeral Muscular Dystrophy Patient Invests $100 Million to Find a Cure
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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…
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Encouraging Findings in Phase 2 Gastric Cancer Clinical Trial
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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,…
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Nanocapsules Enhance Immune Response Against Cancer Tumors
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An individual has a good chance of recovering from an injury to his or her legs or arms, but it is not always the case when the injury involves…
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Scientists Find Biomarker for Predicting Neuron Repair, with Implications for Future Therapies
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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…
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Scientists Demonstrate Gut Microbiome Connection with Transfer of Alzheimer’s to Healthy Animals
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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…
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Promising Interim Data in Phase 1/2 Hunter Syndrome Trial
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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,…
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Wave Life Science’s New Trial Application is Causing Renewed Excitement Around RNA Editing
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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…
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Infantile Spasms: Mom Saves Baby’s Life After Being Dismissed by Two Hospitals
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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…
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Analysis: Reasons for Small Cell Lung Cancer Patients Refusing Treatment
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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…
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Scientists 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…
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The History of Post-Traumatic Stress Disorder
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…
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Researchers Seek Reason for a Higher Survival Rate in Obese Patients with Pulmonary Arterial Hypertension (PAH)
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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…
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Two 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…
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Newborn Screening Saves Lives and Lowers Costs
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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…
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Over 200 Rare Diseases to be Included in Genomics England Research Study
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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…
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FDA Grants Approval for Innovative Dual-Compound Therapy in Pompe Disease
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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…
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The FDA Has Approved the First Blood Test to Identify Multiple Hereditary Cancers
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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…
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Heligenics Finds Variants of IFNB1, Used to Treat Multiple Sclerosis
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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,…
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Death of Muscular Dystrophy Patient Potentially Caused by Adeno-Associated Virus That Delivered Gene Therapy
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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…
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Hairy Cell Leukemia: Drug Combo Achieves Significant Remissions