In a news release from California Polytechnic State University ("Cal Poly"), the University shares how a joint study between itself and the University of South Florida ("USF") has garnered new…
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…
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…
Did you know that zebrafish are often used in genetic research? Not only do they have the same major organs and tissues as humans do, but zebrafish also share a…
The coronavirus pandemic’s effects have wormed their way into every aspect of life, and those of us who spend a lot of time engaging with the medical world know that…
2004 The pain in my right hip had periods of being intense. When the hip became unstable, I decided to visit my ortho. He took one look at it and…
From March 19-22,the Society of Gynecological Oncology (SGO) held its 2021 Annual Meeting on Women's Cancer. Because of COVID-19, the event was held virtually. During the meeting, researchers presented…
Could it be beneficial to harness a potentially devastating virus, such as Zika virus, for use in medical treatment? At first glance, this seems somewhat counterintuitive. Yet a news release…
1967 In 1967 my ortho said "this has to stop." I would just get out of traction; then a cast; heal; then be able to walk for 2 months and…
Timothy and Andrew Revell are two brothers born with Duchenne muscular dystrophy (DMD), a rare neuromuscular disorder that sees the progressive wasting and weakness of the muscles. This condition has…
According to a press release from April 8, 2021, a partnership between Project ALS and the ALS Association has allowed for the development of a Phase 3 clinical trial. Two…
According to a story from Scary Mommy, mother Sara McGlockin recently received devastating news: the drug that was successfully treating her five year old daughter Marian was being discontinued in…
It can take years for patients with rare diseases to get diagnoses and understand more about their conditions. But what if someone developed a new and efficient technology that could…
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,…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Every year, an estimated 3-7 children in Norway are born with phenylketonuria (PKU), a rare genetic disorder characterized by an inability to break down phenylalanine. Researchers in Norway wanted to…
A note from Lisa: Please read and share this piece with anyone living with ALS or any life-threatening condition that can benefit from compassionate use: Lisa’s friend of thirty years…
According to Healio, recently published data suggests that the incidence of scleritis lowered by around 34% over a 22-year period within the United Kingdom. Scleritis, which occurs when the sclera…
Currently, multiple sclerosis (MS), an autoimmune and neurological disorder, is considered either progressive or relapsing-remitting. But could there be additional subtypes? According to new research, yes. As explained in Medical…
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…
I was born in 1960 in Chicago, IL and they knew something was wrong seconds after my birth; what, they had no idea. My mother (I was her 3rd child)…
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 Usher 1F Collaborative has recently announced the opening of registration for its scientific conference titled "Therapeutic Strategies for Large Protein Coding Genes in Usher Syndrome." This event will be…
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…
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