The First Clinical Trial for RNA Editing Begins in the UK
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…
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.
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.…
Neurogeneticist Michael Nalls of the US National Institute on Aging recently told Science Alert that a 2022 study, published in the Journal of Alzheimer’s Disease, completely changed their discovery approach.…
Many helpful guides cover immunology which is the study of the immune system and how the body defends itself against disease. The immune system is comprised of cells, organs, small…
The drug Aficamten is being developed by Cytokinetics as a pill taken once daily for hypertrophic cardiomyopathy (HCM). It is a genetic disease where due to a thickness in the…
In 2023, there have been new drug approvals and increased patient awareness. The American Cancer Society reported a total of 1,958,310 new cancer cases in 2023. Chemotherapy remains the standard…
Carisma Therapeutics, the manufacturer of CT-0508, issued a press release stating that researchers are conducting a combination study using CAR-M cell therapy plus Keytruda (pembrolizumab) in the treatment of overexpressing…
Ferroptosis, a form of cell death, destroys microglia cells, which are associated with the brain’s immune system in vascular dementia and Alzheimer’s. Dr. Stephen Back suggested that attention had not…