The National Institute of Health (NIH) has created a new approach to diagnosing and treating methylmalonic acidemia (MMA), a rare genetic disorder that stops the body from metabolizing fats and…
A healthy brain translates into a healthy blood-brain barrier (BBB) which is a complex series of blood vessels in the brain. When the BBB malfunctions, it may lead to diabetes,…
As seen in the Journal MDPI, chronic myeloid leukemia only got its name in the mid-1800s, when doctors drew together the category based on the occurrence of a “suppuration of…
The rare liver disease primary sclerosing cholangitis (PSC) may impact just .01% of people, but the extremely pernicious disorder's attacks on the liver mean bearers of the disorder account for…
Arimoclomol is a treatment that is in development for ALS, Niemann-Pick disease type C (NPC), and inclusion body myositis (IBM). A recent Phase 2/3 trial has ended in failure, unable…
The FDA has recently provided 89bio with guidance for their upcoming clinical trial of BIO89-100, a treatment for nonalcoholic steatohepatitis (NASH). The trial will advance as planned, beginning in the…
At the American Society of Hematology Annual Meeting in 2020 (ASH 2020), the latest info and data related to Gaucher disease, a form of lysosomal storage disease, was presented. It…
The European Commission (EC) has granted the Orphan designation to Passage Bio for their Krabbe disease treatment, PBKR03. This adds another designation to the medication's list, as it has already…
According to a recent report in Medical Xpress, over twenty studies have indicated escalating events of nonalcoholic fatty liver disease (NAFLD) in children worldwide. In the United States, NAFLD heads…
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…
Orphazyme's latest trial has ended in failure after arimoclomol, a possible treatment of inclusion body myositis (IBM), failed to meet any of its endpoints. As the drug has shown massive…
As Parkinson’s diseases slowly erodes a person’s control of their movement, many normal parts of life become much more difficult. In a new study in Brazil, “Evidence-Based Recommendations for…
Researchers from the University of Barcelona have recently found a new biomarker which may be able to diagnose Parkinson's disease sooner. This biomarker is a protein fragment called GPR37. It…
Sometimes it's hard to pick apart the cause of symptoms. Am I anxious because the bus is late or because Covid-19's insidious shadow is still lingering all these months later?…
Around 30% of all patients with relapsing-remitting multiple sclerosis (RRMS) experience some type of cognitive impairment or decline at the time of diagnosis. Around 50% of patients experience some of…
According to Yahoo Finance, D&D Pharmatech has initiated dosing in their Phase 1 trial of DD01, a treatment for nonalcoholic fatty liver disease (NAFLD) and diabetes in obese patients. As…
Spinal muscular atrophy (SMA) patients aged two months and older will now have an at-home treatment option in Europe. The European Commission (EC) has approved of Evrysdi for those who…
Primary Sclerosing Cholangitis Primary sclerosing cholangitis (PSC) is a rare condition which causes inflammation within the bile ducts of the liver. It can lead to cirrhosis of the liver or…
Financial Burdens in Rare Disease In 2019, 20% of all adults in the United States have reported that they had large medical bills that were unexpected. 18% currently had medical…
Aeglea Biotherapeutics has just announced a new collaboration with Immedica to bring pegzilarginase to other countries outside of the United States. They have created a license and supply agreement to…
In a study published on Hindawi, a team of scientists have discovered that the C-reactive protein to albumin (CAR) ratio can be used to predict the risk of complications following…
Recently, the National Cancer Institute provided a $2.5M grant to support specialized medical research being performed at the Wake Forest Organoid Research Center (WFORCE). According to the press release,…
Recent research indicates that microRNAs may be a potential biomarker to diagnose and judge a Parkinson's patient's prognosis. A study titled “Deep sequencing of sncRNAs reveals hallmarks and regulatory modules…
Just as our emotions control our the body, determining our posture and expression and tensions; our bodies are our feeling devices, with the sensory input and muscle tension in turn…
One year ago, Dr. Audrey Odom John Chief of the Division of Infectious Diseases at Philadephia’s Children’s Hospital, treated a teenage patient for what seemed to be a new disease.…
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