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.
These efforts led to collaboration by the International Brain pH Project that involves 131 scientists in 105 labs from seven countries. The scientists have identified changes in lactate levels…
Continue ReadingScientists have Discovered a Connection Between Metabolic Problems in the Brain and Neurological Disorders
What is primary immunodeficiency? The Mayo Clinic offers this definition: "PI weakens the immune system and as a result infections or other health problems may occur more often. People with…
Continue ReadingWorld Primary Immunodeficiency (PI) Week is April 22-29
Previously, Patient Worthy reported on research focused on the use of a pig kidney in a transplant operation involving a human patient. Richard (Rick) Slayman was released from the hospital…
Continue ReadingMan with Pig Kidney Leaves Hospital, with All Signs Pointing to Success
For decades researchers have had to isolate segments of genes in order to study them. They called the isolated gene a “mini gene.” Ravindra Singh, a biomedical science professor at…
Continue ReadingResearchers Discover ‘Super Minigene’ that Helps Find Potential Therapies for Spinal Muscular Atrophy
Duchenne muscular dystrophy (DMD) is an inherited disease that weakens a child’s muscles and eventually spreads throughout the child’s body. DMD is caused by mutations in the gene that helps…
Continue ReadingGene Therapy Led to “Robust Microdystrophin Expression” in Duchenne Muscular Dystrophy Patient
A new global study (THAOS NCT00628745) of over 6,000 people who had been diagnosed with ATTR amyloidosis found that almost one in every four patients exhibited cardiac and neurological symptoms.…
Continue ReadingATTR Amyloidosis: 1 in 4 Patients Experience Neurological and Cardiac Symptoms
Over 100,000 individuals living in the U.S. are on a waiting list for an organ transplant. An average of 17 people die each day while waiting for a donor organ.…
Continue ReadingDoctors Transplant Genetically Edited Pig Kidney Into 62-Year-Old Man with End-Stage Kidney Disease
During the past three years two drugs (Aduhelm and Leqembi) that were designed to slow the progression of Alzheimer’s disease, have been approved. A verdict by the FDA on a…
Continue ReadingFDA Puts Off Decision on Approval of a New Alzheimer’s Treatment
According to a recent article in MedicalXpress, scientists have discovered a possible method of treating amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The study was also published in Science…
Continue ReadingScientists Find Potential Treatment Approach for FTD and ALS
Neurological conditions now have the highest rates of disability and ill health worldwide. The Disparity Although more than 80% of the neurological deaths and nervous system disorders occur in lower…
Continue ReadingStudy Lists Neurological Disease as the Top Cause of Disability and Illness Around the World
Jenny Decker began her solo voyage in her 1983 Bristol 35.5 Tiama from Honokōhau, Hawaii on June 28, 2023 (see Patient Worthy articles Part 1 and Part 2.) Jenny hopes…
Continue ReadingThis Charcot-Marie-Tooth Disease Patient is Circumnavigating the Globe Solo
A study by a team of scientists at the Children’s Research Institute was reported this week in the journal Nature Communications. The study is welcomed by the medical profession as…
Continue ReadingResearchers Begin Testing Gene Therapies in Whole Human Liver
For years, scientists have attempted to discover what destroys pneumococcal bacteria after a person has been vaccinated. In a recent study, vaccinologists in the U.S., China, and Switzerland, found the…
Continue ReadingAn International Team of Scientists Discovered How Pneumococcal Bacteria is Destroyed After Vaccination
Researchers worldwide spend years studying various aspects of a disease searching for a cure with help initially from public and private sectors. The primary source of the funding is usually…
Continue ReadingMarch is Multiple System Atrophy Awareness Month
A study led by a team of German scientists provides evidence of the safety and efficacy of CD19 CAR T cell therapy and its impact on autoimmune diseases. The study…
Continue ReadingCAR T Delivers Excellent Results for 15 Patients with Autoimmune Diseases
American Journal of Cardiology Source: Association Between Atrial Fibrillation Symptoms and Clinical Outcomes: A Prospective Multicenter Registry Study
Continue ReadingAssociation Between Atrial Fibrillation Symptoms and Clinical Outcomes: A Prospective Multicenter Registry Study
People living with blood disorders like sickle cell disease or thalassemia will now have access to a new blood test that will reduce transfusion side effects. England’s National Health Service…
Continue ReadingThalassemia and Sickle Cell Patients in the UK Gain Access to Blood Transfusion Test to Reduce Side Effects
Cooper Meshew was diagnosed with Charcot-Marie-Tooth (CMT) disease at a very young age. The disorder affects one person in about 2,500 individuals. In fact, Cooper has an extremely rare variant…
Continue ReadingCharcot-Marie-Tooth Disease Couldn’t Stop This Aspiring Farmer
According to information in a recent press release from Wave Life Sciences, dosing has begun in a clinical trial investigating the RNA editing candidate, WVE-006. The drug treats alpha-1…
Continue ReadingThe First Clinical Trial for RNA Editing Begins in the UK
For years, scientists have been working on a completely new concept that generates new neurons in mice with Huntington's disease. They were able to demonstrate that the new cells could…
Continue ReadingHuntington’s Disease: Scientists Hope Their Success in Improving Survival in Mice Will Extend to Humans
Alicia McGrew’s first baby was born eleven years ago. The new parents checked their baby and found him to be “perfect”. Then Patrick, Alicia’s husband, noticed a few small red…
Continue ReadingThese Parents Discovered That All Four Children Had FNAIT
The parents had brought their baby girl home only four days ago when they received an urgent call from the hospital asking them to bring the baby right back for…
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