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1 week ago | 
patientworthy.com | Rose Duesterwald
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 cells being especially vulnerable to treatment at a particular time. Mice models have been successfully treated in the lab while in-human tests will be arranged in the near future.
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2 weeks ago | 
patientworthy.com | Rose Duesterwald
The FDA recently informed that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas. The designation awarded to the experimental drug is an incentive to accelerate the creation of treatments for rare diseases that affect under 200,000 people in the U.S.
IPS Heart’s stem cell therapy is designed to treat heart issues such as generating new heart tissue.
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2 weeks ago | 
patientworthy.com | Rose Duesterwald
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 enteroviruses like a cold virus. The disease exhibits asthma-like symptoms and spreads among people through sneezing and coughing.
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2 weeks ago | 
patientworthy.com | Rose Duesterwald
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 light on an ALS-associated dysfunction that was not known previously. The new research shows that when there is an ALS-linked disfunction in the mitochondria, it can indicate an early sign of ALS, even before other visible signs of the disorder appear.
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3 weeks ago | 
patientworthy.com | Rose Duesterwald
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 disease. In June 2023, Jenny Decker, 40-year-old trauma nurse, began her voyage with a bright and glorious American flag flying off the bow of her 1884 35.5C Bristol named Tiama. Jenny displays Old Glory when she enters foreign ports as a means of identifying Tiama’s country of origin.
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3 weeks ago | 
patientworthy.com | Rose Duesterwald
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 why many of the drugs developed to target Alzheimer’s have not been as effective as promised. As reported by MedicalExpress.com, Ernest Fraenkel, Health Services Professor at MIT’s Biological Engineering Department is the senior author of the new study. Prof.
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1 month ago | 
whatnext.com | Rose Duesterwald
Current treatments have not been effective against cancers of the female reproductive system. The result is that ovarian cancer remains a primary cause of death from cancers that have metastasized (spread). A New DirectionRecently a study identified an essential factor indicating a promising new path for targeted strategies. Targeted therapy, a treatment using drugs to attack cancer cells, differentiates them from healthy cells.
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1 month ago | 
patientworthy.com | Rose Duesterwald
This week, the FDA 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 plaques identified in adults over 55 who have exhibited AD symptoms. This will reduce reliance on PET scans.
According to Dr. Howard Fillit, an AD Foundation science official, over seven million people in the United States are known to have the disorder, and that number is on track to double by 2050.
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1 month ago | 
whatnext.com | Rose Duesterwald
With their findings recently published in Medical Xpress, a team of researchers in Cambridge, UK have identified a treatment approach that has a positive effect on breast cancer survival. Patients were recruited from 23 NHS sites across the UK. Researchers conducting the Partner clinical trial explored the efficacy of combining the drug Olaparib with chemotherapy in the treatment of early-stage breast cancer, especially for patients who had inherited BRCA mutations.
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1 month ago | 
patientworthy.com | Rose Duesterwald
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 to be treated for this rare disorder.
Marley’s family spent years searching for answers but were unable to find any. The family was told Marley’s disorder is rare and disrupts protein production which leads to various neurological and developmental challenges.
