Imagine being Rowan Marshall, a seven-year-old first-grade student with a debilitating rare disease. Rowan made it through the new student formalities. He is doing well at school. Now he faces…
Researchers have been grappling for years with the STAT5 protein as a tool to fight cancer. STAT5 is critical because it is partly responsible for the development and progression…
Before you read on, make sure to read Part 1 and Part 2 of Cristina and Isaiah's story. In Part 1, Cristina discusses Isaiah's GA-1 diagnostic journey and some of her favorite memories of…
After winning the 1998 100m butterfly in Perth, breaking a world record, winning 54 international medals, and becoming the Swimming Hall of Fame inductee in 2022, Michael Klim appeared…
Katie Brown, age 15, loves learning Spanish, swinging, and special effects makeup. In the future, she is considering a possible career as a writer or a special effects artist; she…
It can be difficult to spur interest in rare disease drug development. Forty years ago, the Orphan Drug Act passed as a way to incentivize drug development within this space.…
When Eric and Samantha Rovinetti learned that they were pregnant with their first child, they were overjoyed. They could not wait to welcome baby Adrianna into the world. But shortly…
In June 2022, Andrew Mace received CPR from a neighbor, 21-year-old Alex Duncan, a med student who luckily remembered her instructions while at Cambridge University, UK. Andrew, age 44,…
Before you read on, make sure to check out Part 1 of Cristina and Isaiah's story. In Part 1, Cristina shared the diagnostic odyssey and fight for answers that led to Isaiah's…
Proteinuria refers to excess protein in the urine. In healthy individuals, proteins pass through the kidneys and into the bloodstream. Proteinuria often indicates kidney dysfunction or damage. In fact,…
The failure rate of drug development for Alzheimer’s is 99 percent according to a recent study. Since 1984 scientists have concentrated on developing treatments to prevent the formation of…
Currently, a glioblastoma multiforme (also known simply as glioblastoma or GBM) diagnosis comes with a poor prognosis. There is a 40% survival rate after one year which falls to…
A primary immunodeficiency (PI), or primary immune deficiency disease, is an inherited immune system disorder caused by gene mutations which prevent the immune system from functioning properly. It can…
In the United States, Orphan Drug designation is granted to drugs or biologics being developed to treat, prevent, or diagnose a rare disease. These are conditions affecting fewer than…
At 33 years old, Cristina Urbina has built a beautiful life. Since she was younger, she always knew that she wanted to work with kids. She loves the laughter, the…
Make sure to check out Part 1 of the Schroeder family's story before reading further. CURED Currently, the Schroeder family is doing as well as they can to manage and to ensure…
There have been rising incidences of gastric cancer on a global scale. As a result, this cancer is now considered the fourth leading oncological cause of death worldwide. Gastric cancer…
A Phase 2a study sought to compare the safety, efficacy, and tolerability of ABBV-3373, as compared to adalimumab, for patients with rheumatoid arthritis (RA). Patients involved in the study had…
Silica is a naturally occurring substance that is found in certain types of clay, sand, and rocks. Workers who work with these substances—such as mineworkers, stone masons, construction workers, and…
Before you read on, make sure to check out Part 1 of this story. What is Shwachman-Diamond Syndrome (SDS)? First identified in 1964, Shwachman-Diamond syndrome (SDS) is a rare inherited condition that…
The goal of polymyalgia rheumatica treatment is to reduce inflammation and control or manage the other symptoms. As such, the current standard-of-care includes long-term corticosteroid treatment. According to MedPage Today,…
When asked about the key way that the medical field can better serve patients, Pari Schroeder doesn’t waver: multidisciplinary care. She acknowledges that the medical system can often be very…
Emergency Room (ER) doctors are gradually being replaced by physician assistants and nurse practitioners. The Neiman Institute reported that between 2005 and 2020 the number of ER visits with midlevel…
According to a news release from mid-January 2023, the FDA granted Rare Pediatric Disease designation to SYNB1934 for phenylketonuria. This designation is granted to drugs or biologics being developed for…
A town hall meeting was held on February 7th of this year to discuss designs for clinical trial gene therapies. According to a report in Pharmaceutical-Technology, the FDA experts were…
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