A recent study, titled "Patient and caregiver experiences of living with acute hepatic porphyria in the UK: a mixed method study," investigated the disease burden faced by AHP patients and…
In a study published on Hindawi, a group of patients with severe cystic fibrosis that were awaiting a lung transplant underwent a telerehabilitation program to maintain their lung function. The…
There are many rare diseases that are not yet fully understood, with Parkinson's disease being one of them. Medical professionals have discovered a lot about the underlying causes of the…
CureGRIN is investigating a new symptom of GRIN disorders, a group of rare genetic conditions that cause a wide variety of physical and intellectual symptoms. Referred to as neurostorms, this…
A recent article in Cancer Network announced that motixafortide met its primary endpoint in the GENESIS trial for multiple myeloma patients who were scheduled for stem cell transplantation. Often, the…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to a story from Renal and Neurology News, a recent study has revealed that lupus nephritis, a rare kidney disease associated with lupus, can suddenly recur even years after…
According to a recent article in Parkinson’s News Today, Dr. Per Borghammer, Research Professor at Denmark’s Aarhus University, proposed a new model that has the potential to identify early Parkinson’s…
In a recent study published in the scientific journal Cell Death & Disease, a novel proteasome inhibitor designated D395 was evaluated as a possible treatment for multiple myeloma, a rare form of…
According to a recent article in MedicalXpress, researchers at Houston Methodist have reported acute transverse myelitis (ATM) in forty-three COVID-19 patients across twenty-one countries. Spinal cord lesions developed in these…
A team of great researchers need your help! While the researchers who devised this survey (see below for the link) have had a great response from people with Mal de…
According to MedPage Today, ursodeoxycholic acid significantly improves patient outcomes in relation to intrahepatic cholestasis of pregnancy. This rare liver disorder increases the risk of fetal issues, stillbirths, or preterm…
Sanath Ramesh, a software engineer, created the nonprofit Open Treatments after his son was born with an extremely rare condition without any viable treatment options. Raghav, now two and a…
On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare…
Brickell Biotech has just announced the findings from their Phase 3 study investigating the long-term efficacy and safety of sofpironium bromide gel for the treatment of primary axillary hyperhidrosis. Hyperhidrosis…
The American Academy of Neurology recently held their annual meeting virtually to discuss the latest research in Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS). This meeting was held April 17th through 22nd,…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Bacterial meningitis is a severe bacterial infection that results in inflammation in the brain. Medical professionals are still trying to fully understand this condition, especially how it interacts with the…
On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare…
Most cancers are inflamed (hot tumors) and under the right conditions can be controlled by T-cells. According to a recent article in Genetic Engineering and Biology News, there is another…
TG Therapeutics has been developing ublituximab as a treatment for chronic lymphocytic leukemia (CLL) when used in combination with umbralisib, marketed as UKONIQ. The biopharmaceutical company has recently completed and…
According to a story from Benzinga, there are at least eight drug companies that are currently working on a treatment for Rett syndrome, a rare genetic disorder. With no cure…
According to a story from the Knowridge Science Report, a recent study conducted by the Japanese Kumamoto University indicates that a therapy commonly used to treat diabetes, called metformin, can…
In the last decade, scientists have made great strides in uncovering details of the underlying causes of neurodegenerative diseases. It is only a matter of time that treatment and prevention…
A study published in the Journal of Clinical Oncology has detailed the benefits of different forms of treatment for pediatric patients with adrenocortical carcinoma. These researchers particularly looked at what treatments…
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