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.
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…
Unexpected but positive results from a late-stage clinical trial were announced by Cerevel Therapeutics and reported in BioPharma Dive last week much to the surprise of AbbVie, the company that…
On one side of the equation pharmaceutical companies maintain that if drug prices are restricted it will have a negative impact on innovation. Health economists, on the other hand, maintain…
An American triathlete, Jon Blais, received a diagnosis of amyotrophic lateral sclerosis (ALS) in 2005 when he was 33 years old. The disease is incurable. Jon was told he had…
Exa-cel Gene-Edited Therapy Vertex Pharmaceutical recently announced that Health Canada has accepted its New Drug Submission and granted Priority Review for Exa-cel, a gene-edited cell therapy, for the treatment of…
A recent lab study led to an improved understanding of asthma and its mechanisms of action. The co-senior author of the study, Professor Chris Brightling and his colleagues at the…
Patients with moderate to severe Crohn’s disease have found quick relief from abdominal pain and stool frequency during the first week of induction therapy according to data analysis published in…
Approximately 540,000 individuals in the United States have been diagnosed with autosomal dominant polycystic kidney disease (ADPKD). The disease causes persistent and rapidly accumulating cysts to grow in the patient’s…
These efforts led to collaboration by the International Brain pH Project that involves 131 scientists in 105 labs from seven countries. The scientists have identified changes in lactate levels…
What is primary immunodeficiency? The Mayo Clinic offers this definition: "PI weakens the immune system and as a result infections or other health problems may occur more often. People with…
Previously, Patient Worthy reported on research focused on the use of a pig kidney in a transplant operation involving a human patient. Richard (Rick) Slayman was released from the hospital…
Xstim, Inc. Receives FDA Approval for Xstim™ Spine Fusion Stimulator. (prnewswire.com)
For decades researchers have had to isolate segments of genes in order to study them. They called the isolated gene a “mini gene.” Ravindra Singh, a biomedical science professor at…
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