We’ve reached the month of May, and you know what that means – it is Guillain-Barre syndrome (GBS) and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Awareness Month! During this month, the…
Imagine the feeling of working for years with the goal of saving children who have congenital athymia. Then thirty years later the FDA approves a regenerative medicine based on your…
Alzheimer’s disease is the primary cause of the progressive decline of cognitive function in the elderly. It is characterized by underlying complications in the central nervous system (CNS). To date,…
LPCN 1144 for nonalcoholic steatohepatitis (NASH) has already demonstrated positive results in the LiFT trial, and it is now showing more exciting results in its open-label extension (OLE) trial. Want…
The joy of watching your child grow and meet developmental milestones is not always granted to some parents. Clare’s mother, Allison, described her emotions to CHOP after noticing that Clare…
Because of their rarity, rare diseases often aren't considered or represented in the government. But state by state, we are slowly gaining representation within the legal system. This is being…
For over thirty years, researchers at the University of Pittsburgh (UPMC) have been observing cases of limited scleroderma, a connective tissue disorder that causes thick hard patches of skin. Scleroderma…
A recent article highlighted the struggles of a man in South Yorkshire who has lost multiple jobs due to his diagnosis of cyclical vomiting syndrome (CVS). Cyclical Vomiting Syndrome (CVS)…
How does our body fight infections? After we get infected with a serious illness, in what ways does our immune system prepare? Understanding these processes could help hone treatments and…
Perry Cox is feisty, full of energy, and loves taking part in new activities with her friends. According to her father Griffin, Perry, now five years old, loves dancing and…
According to a recent article, the trial studying the success of CLR 131 to treat Waldenstrom’s macroglobulinemia has shown promising results, allowing it to continue. Waldenstrom Macroglobulinemia (WM) Waldenstrom macroglobulinemia…
Christine Harrison, a grandmother from Poringland, has recently passed away due to progressive supranuclear palsy (PSP). She led a full, rewarding life filled with family and love. She's survived by…
Myotonic dystrophy type 2 is a rare and poorly understood condition. Even less understood is its relation to recurrent ventricular tachycardia, which is even rarer. Myotonic Dystrophy Type 2 Myotonic…
Adolescence is the phase which exists between childhood and adulthood; therefore, people who are between ages 10-19 are typically considered to be adolescents. How does adolescence change the way people…
In 1996, the World Federation of Hemophilia (WFH) launched its Humanitarian Aid Program; it later expanded the program in 2016 to better reach and support patients with hemophilia. The program…
Ala Moshiri from the University of California is both a researcher and an eye doctor. He recently discussed his frustration surrounding the lack of available treatments for so many genetic…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A rare disease is one affecting under…
A new study has examined the long-term impacts of esophageal dilation in eosinophilic esophagitis (EoE). The aim was to uncover the link between dilation and symptom reports. Additionally, the research…
Aiden Collyar, a nine-year-old from New Hampshire, was recently presented with a donation that would allow him to do something he loves: play sports. After he was diagnosed with the…
Have you ever heard of West Nile virus? In many cases, this mosquito-borne illness does not cause severe issues. However, in just about 1% of cases, West Nile virus can…
According to a recent article, medical professionals are urging people to be careful and avoid tick bites as May is Lyme Disease Awareness Month. Lyme Disease Lyme disease is a…
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 process of an organ transplant is a long one, and I'm not just talking about the procedure itself. For many patients, the majority of the time is spent waiting…
During the Phase 1/2 SUNRISE clinical trial, which evaluated LB-001 for pediatric patients with methylmalonic acidemia, at least two participants experienced serious and severe adverse reactions: thrombotic microangiopathy. While investigating…
According to a recent article, Krishnansu S. Tewari, MD, discussed using bevacizumab, niraparib, and olaparib in patients who have received and completed chemotherapy for their advanced ovarian cancer. Ovarian Cancer…
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