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.
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…
According to a recent American Society of Clinical Oncology (ASCO) press release, Dr. Jason Westin provided testimony regarding a nationwide shortage of cancer drugs, numbering more than 15 on the…
The treatment of young patients with R/R acute myeloid leukemia (AML) poses a challenge for clinicians. Chemotherapy regimens, such as high doses of cytarabine, fludarabine, and other salvage reduction therapies,…
A recent release published in BioSpace announced that a gene therapy company is vigorously pursuing the study of AVB-101 to treat patients with frontotemporal dementia (FTD), including FTD with GRN…
In 2013, the Morells welcomed two identical baby girls, Kaylie and Kenzie. The joy they felt soon disappeared as the twins began to slip back from their normal infant and…
Takeda presented encouraging results from the Phase 2b trial (NCT05153148) at the November 2023 American College of Rheumatology (ACR) convergence in San Diego. The Phase 2b study evaluated TYK279 which…
Bladder cancer patients and their families have faced years, and even decades, of waiting for a breakthrough in bladder cancer treatment. For some patients, the results of two clinical…
A post hoc analysis of CIMZIA (certolizumab pegol) compared to the drug adalimumab for treating rheumatoid arthritis (RA) patients was presented at the November 2023 ACR Convergence in San Diego.…
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