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.
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…
Duchenne muscular dystrophy (DMD) is an inherited disease that weakens a child’s muscles and eventually spreads throughout the child’s body. DMD is caused by mutations in the gene that helps…
A new global study (THAOS NCT00628745) of over 6,000 people who had been diagnosed with ATTR amyloidosis found that almost one in every four patients exhibited cardiac and neurological symptoms.…
Over 100,000 individuals living in the U.S. are on a waiting list for an organ transplant. An average of 17 people die each day while waiting for a donor organ.…
During the past three years two drugs (Aduhelm and Leqembi) that were designed to slow the progression of Alzheimer’s disease, have been approved. A verdict by the FDA on a…
According to a recent article in MedicalXpress, scientists have discovered a possible method of treating amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The study was also published in Science…
Neurological conditions now have the highest rates of disability and ill health worldwide. The Disparity Although more than 80% of the neurological deaths and nervous system disorders occur in lower…
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