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.
Researchers have discovered a potential “window” whereby babies born with inherited diseases may be treated with gene therapy sent directly into their circulatory systems. The window consists of circulating stem…
The FDA recently informed Muscular Dystrophy News that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas.…
In 2014 the U.S. experienced a nationwide outbreak of EV-D68, raising concerns about children’s respiratory health. Acute flaccid myelitis disease (AFM) also spiked. EV-D68 is defined as a group of…
Researchers at the UK Dementia Institute in London (UK DRI) have identified a common denominator for various gene mutations that cause amyotrophic lateral sclerosis (ALS). A new CRISPR study shed…
As many Patient Worthy readers are aware, Jenny Decker, a Charcot Marie Tooth patient, has made a serious commitment to circumnavigate the globe solo despite an incurable and eventually fatal…
A new study by MIT researchers reveals new potential targets such as cellular pathways and genes that have not been previously linked to Alzheimer’s. These results may provide answers to…
This week, the FDA approved the first blood-based diagnostic test for Alzheimer's disease (AD). The test, known as "Lumipulse G pTau 217/β-Amyloid 1-42 Plasma Ratio," is designed to detect amyloid…
In 2023, Marley Mansour of Rowlett, Texas and her family were told that Marley had an extremely rare genetic condition called NARS1 disorder. Now, Marley is the world’s first person…
The results released from the Phase III ESSENCE clinical trial, and recently published by Medical Xpress and in the NEJM, found that the drug semaglutide is effective in the treatment…
Raiden Messerli and his family thought that he had an ordinary case of the flu. Brandi Lewis, Raiden's mother, discussed in an interview with KCCI8 News how she still vividly…
This article is a continuation of a previous story, and is an excerpt from original author Isabella Cristobal's post. To read the first part of the article, please click here.…
Professor Ronald Breslow was a chemist at Columbia University who, in 1958, proposed the theory that vitamin B1 (thiamine) could regulate metabolism pathways in the body by stabilizing a reactive…
Immune dysfunction and inflammation have been found to play significant roles in Alzheimer's disease. An analysis of brain tissue in an animal model reveals a potential for immune system dysfunction at…
Current research indicates that children and adolescents who were unvaccinated during the pandemic were much more likely to develop Long COVID. Researchers led by Perelman Medical School at the University of…
Humans have been using tobacco as a drug for more than 12,000 years. It wasn't until the early-20th century that the link between tobacco and lung cancer (and other health problems)…
Blunt trauma from car accidents contribute to optic nerve damage and blindness. Glaucoma is also responsible for similar optic nerve damage. It is known to be the second worldwide cause…
In 1999, Towana Looney, a resident of Alabama, donated one of her own kidneys to help save her mother’s life. Then Towana waited eight years for an organ transplant that…
A recent study by Texas A&M researchers evaluated the results to male offspring of parental alcohol exposure. The exposure to the chemical diethylnitrosamine was found to be disruptive to mitochondrial,…
Due to the efforts of public health officials in the 1940s, Murine typhus was almost eradicated in the United States. However, there now appears to be a resurgence in the…
Malaria remains responsible for 50,000 deaths annually. An ever-expanding population coupled with malaria gaining resistance to treatments are prominent roadblocks. As reported in Science Alert, the plan is radical. The…
Prader-Willi syndrome is a leading cause of obesity in children. The rare disease was discovered over seventy years ago, yet this is the first therapy to be approved by the…
National Cancer Center researchers have discovered a mechanism for liver regeneration in animal models triggered almost immediately after episodes of severe liver damage. The study, reported in MedicalXpress, explains that…
Professor Sophia Padilla, lead author of a new study on antibiotic resistance describes the cycle in an article published in UC Irvine’s American Chemical Society Journal. Scientists develop drugs to…
Scientists have earnestly delved into a new study to test a drug for individuals who have genetically inherited a type of early-onset Alzheimer’s disease known as “dominantly inherited Alzheimer’s disease”.…
One Million People: That is an estimate of the number of individuals who die from infections that resist drugs such as antibiotics according to a report in BBC News. Yet,…
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