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.
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…
A Baylor College team of researchers headed by author and Associate Professor of Pathology, Liaising Yen, have spent over ten years working on a technology to effectively regulate gene expression.…
The Griffins of Harrisburg, North Carolina enrolled their son in the AGAMREE clinical trial in 2016. Their son was the first participant in the trial studying the investigational drug vamorolone…
Sarepta Therapeutics’ treatment Elevidys (delandistrogene moxeparvovec-rokl) received conditional approval from the FDA in June 2023. The designation implies that if the drug is used in accordance with its label, it…
Controversial Alzheimer's drug comes off the market (axios.com)
The immune system is constantly on the alert for cells that have sustained DNA damage. When a cell divides its DNA molecules are, to varying degrees, usually damaged. Yet researchers…
The agency in Guangzhou, China, that arranged three-year-old Shelby’s adoption by a family in the US disclosed to Michelle and Adam Campbell that Shelby had beta thalassemia disorder. Yet, that…
Eliza is now 13 years old and was diagnosed at the age of three with a neurodegenerative disorder known as Sanfilippo syndrome. Eliza’s mother, Dr. Cara O’Neill, a pediatrician and…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
January 24, 2024 is recognized as Moebius Syndrome Awareness Day, a day to spread awareness about the rare disease Moebius syndrome among the medical field and the general public. The…
Macrophages play a crucial role in responding to injury or infection. Thomas Tapmeier, a researcher at Monash University and Oxford University, recently discussed a study he co-authored in MedicalXpress.…
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