Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In 2015, the United States approved Kanuma (sebelipase alfa) for the treatment of infants living with rare lysosomal acid lipase deficiency. However, the therapy was not approved in other countries;…
According to a story from Healio, a recent study has found that use of hydroxyurea in alignment with treatment guidelines has the potential to bring down symptoms of cerebrovascular disease…
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.…
An estimated 90% of the global population is infected with Epstein-Barr virus (EBV). In some cases, this infection is asymptomatic; in others, EBV can lead to numerous health impacts, including…
Michael Strahan is well-known for being an American television personality, journalist, and former NFL Defensive End (DE) for the New York Giants. Now, the family man and proud father is…
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…
In 2021, approximately 37 people per every 100,000 were diagnosed with idiopathic hypersomnia (IH), a neurological disorder characterized by excessive sleepiness and excessive daytime sleepiness. This amounts to roughly…
As it stands today, there are no cures for CDKL5 deficiency disorder. Treatment is symptomatic and supportive, and includes physical, occupational, and speech therapy. CDKL5 deficiency disorder causes seizures,…
AIV001 (axitinib): a potential, non-surgical therapeutic option for people with basal cell carcinoma (BCC). AiViva Biopharma describes AIV001 as: a novel formulation of a multi-kinase inhibitor combined with AiViva’s…
In Gibson City, IL, students at Gibson City-Melvin-Sibley High School (GCMS High School) are fighting to find a cure for Friedreich’s ataxia (FA). According to reporting from Jared Thomas…
According to a story from Healio, Rohit Kohli, MBBS, MS, division chief of gastroenterology, hepatology and nutrition at Children’s Hospital Los Angeles, spoke at the World Congress on Insulin Resistance, Diabetes…
Nathan Ehrlich, age 40, has had more health troubles than the average person, to say the least. As a teenager, he was diagnosed with leukemia. Thanks to the support of…
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…
The 66th American Society for Hematology (ASH) Annual Meeting and Exhibition took place from December 7-10, 2023. During the meeting, researchers and other stakeholders shared insights into clinical trends,…
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…
In the United States, Breakthrough Therapy designation is granted by the U.S. Food and Drug Administration (FDA) to expedite drug development and review. This designation is granted to therapies that…
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…
Healio Psoriatic Disease recently reported that Vyjuvek (beremagene geperpavec-svdt), a topical gene therapy designed for people with dystrophic epidermolysis bullosa (DEB), was granted Orphan Drug designation (ODD) by the Japanese…
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 story from Science Daily, a team of scientists affiliated with the Salk Institute recently published a study on December 6, 2023 focused on how a class of…
In March 2022, the U.S. Food and Drug Administration (FDA) decided against the approval of oleogel-S10 for epidermolysis bullosa (EB); the FDA noted that, prior to approval, it would need…
In an investor news release from December 2023, Moderna and Merck shared follow-up data from the Phase 2b KEYNOTE-492/mRNA-4157-P201 clinical trial. During the trial, researchers explored the impact of Keytruda…
In December 2023, Merck and HiberCell announced that the organizations would be partnering on a Phase 1b clinical study to examine a drug combination for clear cell renal cell carcinoma…
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