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.
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…
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…
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…
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…
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…
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…
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…
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.…
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…
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…
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…
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…
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…
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).…
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…
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…
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…
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…
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…
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…
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…
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,…
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…
Patients Aged 80 to 90 Respond to Standard-of-Care AML Treatment | Inside Precision Medicine
Angelman syndrome begins at birth and although symptoms vary, most children exhibit common developmental delays such as: • Infants showing an inability to support their head • Unable to pull…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
