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.
Each summer advocates for rare diseases take advantage of longer daylight hours and use the opportunity to showcase the hopes, needs, and wants of people with rare diseases such as,…
The success of the CARES 310 Trial Phase III (NCT03764293 ) was reported this week in Biospace by Elevar Therapeutics. The combination of two drugs, rivocera, and camrelizumab proved to…
According to an article in MedicalXpress News Today, current research indicates that rare diseases in the United States affect 25-30 million people. The numbers keep rising and rival diseases…
The long-awaited decision by the European committee CHMP was announced recently by Amylyx Pharmaceutical (the Company) based in Cambridge, Massachusetts just as anticipated. The EMA issued its negative opinion of…
Professor Timothy Yu developed the custom drug milasen named in honor of Mila, an 8-year-old girl with Batten disease. The drug is the first drug specifically designed for one…
Lonnie's mother, Stephanie Pesterfield of Colorado Springs, still finds it difficult to talk about her son Lonnie’s near-death experience. Yet as published recently in People, Stephanie and her son want…
Leukemia is cancer that begins in the bone marrow and involves abnormal white blood cells (WCB) from lymphatic tissues and bone marrow. Scientists have new theories as to why certain…
Prothena Corporation which is based in Dublin, Ireland recently announced, via Business Wire and Biospace results of the VITAL Phase 3 clinical trial showing a significant survival benefit…
Findings from the Max Delbrück Center and the Charité Universitätsmedizin Berlin have been published in the European Respiratory Journal stating that a triple combination therapy can improve CF symptoms…
According to a report in the morningstar.com, the FDA has approved AstraZeneca/Sanofi shot that protects infants and toddlers against RSV. The 2022/2023 winter saw an RSV increase, especially among…
Dr. Brooke Molina and co-authors at Pittsburgh University have reassuring news for parents of children with ADHD. The team conducted an in-depth study, recently published in JAMA, that followed…
Most professional athletes are able to cope with occasional sprains and sore muscles. Claire Fahey a professional sports player, says she could brush off most of the inconveniences that came…
American consumers have access to the most advanced pharmaceutical systems in the world. Recent medical and technical advances seem to support that reputation. According to Medical Xpress, a Swiss…
The FDA has approved Rystiggo, a developed by UCB in Brussels, to treat two types of myasthenia gravis. The approval was based on the results of the Phase III…
The incidence of gastroschisis has doubled over the past 20 years. Read “Jeanie’s Gastroschisis Baby” and learn from a brave, young first-time mom about discovery and how Gastroschisis can be…
Dr. William Lynes completed his residency in 1987 at Stanford University. He began his practice at the Kaiser Permanente Riverside Medical Group in California. It was the idyllic life that…
It looked as if the fate of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy was pretty much sealed until an FDA official intervened. This information was provided by…
BCMA is a protein that is found in multiple myeloma cancer cells. CAR-T cell therapies such as Carvytki are engineered to home in on BCMA proteins. According to a…
According to a recent article in the Pharma Times, E02463 is a therapy for indolent or slow growing, not yet problematic non-Hodgkin B cell lymphoma (iNHL). Enterome, its developer,…
According to a recent article in People Magazine, this Illinois family has a dream: to turn amyotrophic lateral sclerosis (ALS) from a fatal to a chronic illness in Brian’s…
If you’ve been there, then you know. Patients experiencing cancer, especially the rare aggressive types such as pancreatic cancer, and requiring targeted treatment for the disease may soon be…
The five-year rate of survival for pancreatic cancer is at 12% having the highest mortality rate of any major cancer. Defence, an aptly-named biopharmaceutical company in Vancouver, B.C. recently…
A recent article published in GlobeNewswire captured the results of the trial that was presented at the 2023 Annual ASCO Meeting from June 2nd to June 6th, 2023 in Chicago.…
At ASCO’s 2023 convention for patients diagnosed with glioma and a specific genetic alteration, Servier Pharmaceuticals announced that vorasidenib, the drug being investigated in its Phase III trial (INDIGO)…
The FDA advisory panel which is composed of specialists who advise the FDA on brain medications, recently voted 6-0 in agreement on that the new drug, Leqembi, was effective…
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