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…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
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…
Have you ever heard of Orphan Drug designation? This designation, granted by the U.S. Food and Drug Administration (FDA), is given to drugs or biologics that are designed to treat,…
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,…
According to a story from BioPharma Reporter, the pharmaceutical company Candel Therapeutics recently announced that its investigational therapy designated CAN-2409 has earned Fast Track designation from the US Food and…
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…
GRIN2B Disorders are rare changes in the GRIN2B gene, some causing gain of function, some loss of function, and others unusual neurotransmissions, but all resulting in a variety, and a…
Right now, there are minimal therapeutic interventions for people living with idiopathic pulmonary fibrosis (IPF). Because of this, IPF comes with a life expectancy of 3-5 years past diagnosis.…
Clinical trials are helpful tools in deepening our understanding of certain diseases and evaluating and identifying effective treatment options. Unfortunately, these trials don't always garner the necessary information. For…
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