Funding for clinical trials can be an issue, especially for rare disease studies. Luckily, there are programs that exist to help with the financials, such as the "Clinical Studies of…
Recently, biopharmaceutical company PTC Therapeutics, Inc. performed a five-year data analysis to evaluate the continued safety, efficacy, and tolerability of PTC-AADC. The company developed this novel gene therapy to improve…
According to Healio, the FDA recently granted Orphan Drug designation to nadunolimab (CAN04), a treatment candidate for patients with pancreatic cancer. Orphan Drug designation is given to drugs or biologics…
Patient Worthy recently had the privilege to view a selection of short films about rare disease at Biotech Week Boston. These films highlight the stories of families and patients as…
A recent study conducted by the Penn State College of Medicine revealed that taking glucagon-like peptide-1 receptor (GLP-1R) agonists six months before COVID-19 infection decreases the risk of a severe…
A new collaboration between the University of Texas's MD Anderson Cancer Center and the Rare Cancer Research Foundation aims to solve a problem that has been a major obstacle for…
When we are faced with a limiting health issue where performing “simple” everyday activities becomes a challenge, our self-confidence and self-worth can dwindle. This can happen with any health condition…
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 current standard-of-care for children with cerebral palsy often consists of a blend of occupational and physical therapy. But what if another therapy could be more beneficial? Medical XPress describes…
Long QT syndrome (LQTS) is a rare condition that's characterized by fast and chaotic heartbeats. Episodes have the potential to cause fainting, seizures, or even death. Because of this, affected…
If you've ever heard any discussions around the topic of opioids, you probably know that there is a lot of controversy and a similar deal of complexity. However, researchers in…
Kristin woke up the morning of her 19th birthday with a startling realization: she was unable to speak. It was hard to describe the feeling, but when she tried to…
What would you do to support your child? Would you take on a daunting 100 kilometer (62 mile) run? According to the Daily Record, a mother and her friends in…
In the past, there have not been any available treatments to tackle cholestatic pruritus, or a severe and unrelenting itch, for patients with liver diseases such as Alagille syndrome. However,…
NOTA, The Network of Tyrosinemia Advocates, held their 4th Annual NOTA Experience, a family conference in September, despite the extraordinary constraints of time, a pandemic, transportation challenges, and the special…
Clinical trials are important to understanding both more about certain conditions and more about the safety, efficacy, and tolerability of drugs designed to treat these conditions. Currently, biopharmaceutical company AMO…
It's a beautiful thing when a community pulls together to support one of its members. This is exactly what the Madeira community is doing for 16-year-old Diego Ramirez. He recently…
Lytle, Texas is a small town located outside of San Antonio. Recently, the entire community came together to support a young boy living with autosomal recessive polycystic kidney disease (ARPKD).…
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…
The FDA created its Orphan Drug Act to incentivize drug developers to develop solutions for patients with rare or life-threatening conditions. Now, Orphan Drug designation is granted to drugs or…
Alan Jackson has been a big part of the country music scene for a long time, with songs like Remember When and Livin' On Love. Recently, he revealed to his…
In the past, caffeine has been somewhat of a complex or even controversial topic. While some research shares that it is bad for health, others laud caffeine’s ability to enhance…
In the United States, Orphan Drug designation is granted to drugs or biologics which show promise in treating patients with rare and life-threatening conditions. For the purpose of this article…
Science Direct recently published a Respiratory Medicine Case Report that may impact future Cystic Fibrosis (CF) treatment. The case involves adult brothers herein referred to for purposes of this…
Dave and Kendra Riley have a beautiful family: them and their daughters Eva, Kiera, and Olivia. Yet the family has faced some scary and difficult obstacles over the last…
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