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.
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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…
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Granulomatosis with Polyangiitis: Removal of Wisdom Teeth Eventually Led to a Lung Transplant for this Teen
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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…
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Scientists May Have Found a Possible Cure for Acute Leukemia in Base Edited T-Cells
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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…
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Phase 3 Trial Shows Significant Benefit in the Treatment of AL Amyloidosis
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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…
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Two Studies Find that a Three-Drug Combination Improves Cystic Fibrosis Symptoms
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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…
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FDA Approves New RSV Treatment to Protect Infants and Children up to 24 Months
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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…
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ADHD: Are Prescribed Stimulants for Children Associated with Future Substance Use?
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…
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She Has Lupus But Just Won Her 7th ‘Real Tennis’ Title and Remains a World Champion
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…
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Study: Recent Drug Approvals Offer Less Therapeutic Value
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…
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The FDA Approves New Myasthenia Gravis Drug That Targets the Two Most Common Variants
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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…
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Celebrate Gastroschisis Month with Jeanie and Her Gastroschisis Baby
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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…
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From Physician to Survivor: A Journey Through Burnout, Mental Illness, and Triumph
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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…
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A Top FDA Official Overrules Reviewers’ Rejection of Sarepta’s DMD Gene Therapy
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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…
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J&J’s Therapy Shows Early Success Against Multiple Myeloma
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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,…
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Enterome Announces a Positive Profile for Indolent Non-Hodgkin Lymphoma
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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…
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Documentary: ALS Patient Starts New Awareness Campaign “I AM ALS”
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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…
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‘Smart Drug’ for Pancreatic Cancer Being Evaluated in Telehealth Clinical Trial
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…
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ARM Anti-Cancer Vaccine Begins Testing Against Pancreatic Cancer
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.…
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Positive Results in Cancer Vaccine Trial for Merkel Cell Carcinoma and Cutaneous Squamous Cell Carcinoma
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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)…
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ASCO 2023: Vorasidenib Hailed As “Unprecedented Improvement” in Glioma Treatment
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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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FDA Panel Backs Approval of a New Drug for Alzheimer’s
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Patients who have been heavily pretreated for relapsed/refractory multiple myeloma participated in the phase 2 study of bispecific T cell redirection antibodies. The participants responded to treatment with a combination…
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Bispecific T-Cell Redirection Antibodies in the Treatment of Multiple Myeloma
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June 17th is International CDKL5 Day. During the month of June let’s tag every day with a reminder to those in the CDKL5 community to promote collaboration and fundraising.…
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June 17th is International CDKL5 Day
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Effective June 1, 2023, the FDA lifted its clinical hold on Foghorn Therapeutics’ Phase 1 study of FHD-286 dose escalation in patients with acute myeloid leukemia and myelodysplastic syndromes. The…
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FDA Lifts Clinical Hold on Phase 1 AML and MDS Clinical Trial
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An observational study of 599 survivors of COVID-19 was conducted at the Papa Giovanni Hospital in Bergamo, Italy three months after hospital admission. The patients were evaluated for thyroglobulin…
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Could COVID-19 be a Cause of Autoimmune Thyroid Disease?
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Relyvrio was officially approved by the FDA for treating ALS in September 2022. However, according to an article in BioPharma Dive, there was considerable controversy surrounding the clearance of the…
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ALS Drug Facing Possible Rejection by the European Medicines Agency