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.
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…
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.…
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.…
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…
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…
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…
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…
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…
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…
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…
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…
BioPharma Dive Source: Regenxbio data suggest ‘niche’ in Duchenne gene therapy
BioPharma Dive Source: Amylyx ALS drug fails crucial study, putting company’s future in doubt
American Journal of Cardiology Source: Association 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…
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…
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…
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…
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…
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…
Amyotrophic lateral sclerosis (ALS), ravages nerve cells in the spinal cord and brain. It is at times called Lou Gehrig’s disease, named after the famous baseball player who died of…
Researchers have demonstrated renewed interest in vaccines that treat Alzheimer’s. The treatments remove toxic proteins from the brain. The author of a recent article in Reuters interviewed ten researchers and…
Out of 103,000 people in the United States waiting for a transplant, almost 88,000 individuals on the waiting list need a kidney transplant. These statistics were recently provided through Organ…
Prilenia Therapeutics, Waltham, Massachusetts and Naarden, Netherlands is a biotechnology company with a focus on developing therapeutics to treat neurodevelopmental disorders and neurodegenerative diseases. Prilenia presented data this month at…
The primary outcome from the Phase III NETTER-2 clinical trial as reported recently in Globe Newswire, was heralded as extending progression free survival from 8.5 months to 22.8. The participants…
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