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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Researchers at Washington University’s medical school have used a new technique to study brain neurons in a lab setting, allowing them to bypass the need to rely on a brain…
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The First Successful Attempt to Study Alzheimer’s Disease Using Lab-Derived Neurons
At Japan’s Fujita University, researchers reported to Inside Precision Medicine that a new risk measurement may add a layer of accuracy in predicting sudden cardiac death. The measure, called estimated…
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Researchers Identify Kidney Disease as a Sudden Cardiac Death Risk Factor
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On July 11th Dr. Mathew Winton went to Capitol Hill with senators and staff from both parties to discuss collaboration with the FDA regarding the pending termination of the Priority…
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The Urgency of Reauthorizing Rare Disease Priority Review Vouchers
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The long-awaited positive results of Annexon Inc.’s Phase 3 trial investigating ANX005 for Guillain-Barré syndrome (GBS) was presented at the PNS 2024 Annual Meeting. Several leading global GBS experts spoke…
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Positive Results from the Phase 3 Trial of ANX005 in Guillain-Barré Syndrome
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Recently there has been an increasing amount of research attempting to control CRISPR gene-editing systems. These systems are found in many types of bacteria as a defense against viruses. The…
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More Compact CRISPR Protein Opens up Gene Therapy Options
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Shanghai's Junshi Biosciences Co. (The Company) recently announced in Biospace, that its product, toripalimab (TUOYI®), combined with bevacizumab as a first-line therapy for hepatocellular carcinoma (HCC), met its endpoints in a…
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Toripalimab Combined with Bevacizumab Meets Primary Endpoint for Advanced Hepatocellular Carcinoma
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Ocugen, Inc., a biotechnology company based in Malvern, Pa. recently reported to BioSpace that DSMB gave a favorable review of its Phase ½ ArMaDa clinical study of OCU410. Approximately 10…
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Geographic Atrophy Trials Continue to Make Progress
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Individuals who appear to have Long COVID may exhibit a variety of symptoms lasting weeks or even years. The latest estimates are that about 10-20% of people infected with SARS-CoV-2…
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Long COVID: What Have We Learned?
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A team of scientists from Japan appear to have pioneered a method for reversing synapse damage in an Alzheimer's disease mouse model. Although encouraging, the theory must be tested in…
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Japanese Scientists Reversed Signs of Alzheimer’s by Restoring Synapse Function in Mouse Model
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The idiom ‘third time’s a charm’ may have some significance in this instance. The panel that advises the FDA just voted unanimously in favor of Eli Lilly’s experimental drug, donanemab.…
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FDA’s Panel of Experts Vote 11-0 in Favor of Donanemab to Treat Alzheimer’s Disease
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Professor Michael Goran was referring to research data that was presented at the annual 2023 conference of the Hepatology, Gastroenterology, and Nutrition Society of North America when he stressed that…
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Higher Sugar Intake Boosts Risk of Liver Fibrosis in Latino Teens
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Ruud Dobber, executive VP of biopharmaceuticals at AstraZeneca was quoted in a press release dated saying that type 2 diabetes is on the rise in adolescents and younger children with…
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Farxiga® has been FDA Approved to Treat Children and Adolescents in the U.S. for Type 2 Diabetes
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Pfizer has recently paused its Phase III clinical trial that has been assessing the Duchenne muscular dystrophy candidate fordadistrogene movaparvovec. The Phase II DAYLIGHT trial (NCT05429372), enrolled ten boys ages…
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Pfizer Acknowledges the Death of a Young Boy Who Died One Year After Participating in its Phase II Clinical Trial to Treat DMD
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One year ago, Jenny Decker age 40 began her journey around the world sailing 3,200 miles to her first stop in Fiji. She reported to Practical Boat Owner that she…
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Jenny Decker May Have Charcot-Marie-Tooth Disease, but She is Sailing Around the Globe
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This week Neurocrine Biosciences announced the results of its KINECT®-4 Phase 3 study of INGREZZA® capsules. Results of the trial were published in the Journal of Clinical Psychopharmacology. A long-term…
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Tardive Dyskinesia: Phase 3 Study Results Indicate Improvement with Use of INGREZZA®
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Cartesian Therapeutics, Inc., a biotechnology company, recently announced that the FDA has awarded the Regenerative Medicine Advanced Therapy (RMAT) designation to its lead product, Descartes-08, to treat myasthenia gravis (MG).…
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Cartesian Therapeutics Earns Regenerative Medicine Advanced Therapy Designation for Myasthenia Gravis
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Occasionally the acronym NEGU may appear in a social media post. It has been established as a rallying cry and support for people with cancer or other debilitating diseases. It…
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Karen Overpowered Dystonia and Passes the Freedom, Like the Olympic Flame, to her Readers
BioSpace recently published a press release discussing the interim results highlighting a Coya Therapeutics’ study. The study evaluates the safety, tolerability, and biological activity of LD IL-2 in 38 patients…
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Encouraging Results from a Phase 2 Study of IL-2 to Treat Alzheimer’s Patients
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Jianmin Fang, M.D. the CEO of the biotechnology company RemeGen, recently announced in PRNewswire that the company is continuing its significant progress in its research of Telitacicept (RC18) to treat…
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Enrollment Completed in Clinical Trials for Primary Sjögren’s Syndrome and IgA Nephropathy
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A major illness can rob some people of their strength and courage, but in other cases it motivates people to get help and, if possible, to help others. 20 years…
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She Has Cervical Dystonia but Will Wing Walk on a Biplane to Raise Awareness
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People who have bipolar depression face a 50% lifetime risk of attempting suicide and a 20% risk of death from suicide. Over seven million people in the U.S. are struggling…
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Suicidal Bipolar Depression: Clinical Trial Results of NRX-101 Found Superior Safety and Similar Efficacy Compared to Lurasidone
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In a clinical trial conducted by Dr. Talal and his colleagues, the team compared hepatitis C response rates for 602 patients with opioid use disorders. The trials took place…
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Telemedicine Programs Improve Cure Rates for Hepatitis C vs. Specialist Referral
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As reported in Biopharma Dive, the FDA’s decision to strengthen its authority involving laboratory developed tests (LDTs) has drawn considerable objections from various healthcare groups. However, the Agency has held…
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New FDA Regulation on Laboratory Developed Tests Draws Objections from Healthcare Groups
You may not have heard of spinocerebellar ataxia 4 (SCA4). It is rare and it is a devastating movement disease. SCA4 generally begins when a person is 40 or 50,…
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ICYMI: After a 25 Year Search, a Doctor Discovers the Cause of Spinocerebellar Ataxia 4
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The ALS space looked promising in 2022 after Relyvrio’s approval and a green light for Oalsody in 2023. Then progress stalled a bit in 2024 as a result of Relyvrio…
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Amyotrophic Lateral Sclerosis (ALS): Where Are We Now and Where Are We Going?