Thinking about the year-end holidays brings up a kaleidoscope of images, and an array of emotions for me. And everyone else too, I imagine. There are the vivid memories of…
Did you know that zebrafish, a tropical freshwater fish, are being more commonly used to model and study human diseases? Now, says Phys.org, researchers from the Garvan Institute of Medical…
Did you know The Orphan Disease Center (ODC, a center within the Perelman School of Medicine at the University of Pennsylvania) has a program to assist patient groups and families…
When Camden Rau was born, his parents immediately knew that he was facing some sort of health problem. According to Camden's father, Steven, who spoke to Wink News, Camden's skull…
Ever since he was a young boy, JR Trout, who lives with spinal muscular atrophy (SMA), had a sense that there was something different about him. For years, he never…
Adam Settle, is an artist, football lover, and the youngest of eight. He’s also the subject of the new book, No Day Wasted: The Adam Settle Story, by Susan F. Stallings.…
From December 1-4, 2020, Reuters hosted a virtual conference called Pharma & Patient USA 2020. The primary focus of this event was furthering patient centricity in disease development as well…
Obesity is considered to be excess fat accumulation that may lead to health issues. Worldwide, the prevalence of obesity has greatly increased over the years; the World Health Organization (WHO)…
The Orphan Drug Act originated in 1983 as an incentive for pharmaceutical companies who worked on developing treatments for rare disease. Since rare diseases are well, rare, choosing to work…
The latest news from Israeli scientists, published in the Times of Israel, is that researchers at Tel Aviv University have accurately targeted cancerous cells in mice using CRISPR.…
On November 19, 2020, Research!America hosted an informational webinar titled "From Nobel Winning Science to Next Generation Treatment: Tracing the Path of a Rare Disease Breakthrough using RNAi." This webinar focused…
According to a report in BioPharma Dive, Brainstorm Therapeutics, a New York biotechnology company, said that while its NurOwn therapy for amyotrophic lateral sclerosis (ALS) appeared to have a…
There is a certain time of year in which the trees change color, the air becomes crisp and we pour gravy on everything. As I am typing this, I am…
PMDD Premenstrual dysphoric disorder (PMDD) is a psychological condition which cyclically occurs with the menstruation cycle. Unfortunately, because the symptoms of this condition can mirror many mental health diagnoses, it…
Navigating a Rare Disease in a Pandemic The COVID-19 pandemic has affected everyone, but those already living with a rare disease have unique challenges. With a compromised immune system, they…
According to a story from PR Newswire, the pharmaceutical and diagnostics company The Menarini Group recently announced that its drug tagraxofsup (marketed as Elzonris) has a received a positive opinion…
Robert is a 43 year old father of a two year old son named Parker. Robert was diagnosed with inoperable stage 4 bladder cancer about a year and a half…
We often hear people talking about the stress they are feeling during the holidays, while those in the rare disease community silently think, "You have no idea!" During this season,…
"Repurposing" drugs means that researchers may discover that certain compounds or therapies for one condition could be translated into the treatment plan for other conditions. Recently, research published in the American…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy, or SMA, is a genetic disease that affects an individual’s central and peripheral nervous systems, as well as their…
Rhythm Pharmaceuticals, based in Boston, MA, is a biopharmaceutical company focusing on the development of therapies to treat rare genetic diseases that cause obesity. Until now, there have not been…
IntraBio has recently launched an extension study of their Phase II trial of IB1001, a treatment for Niemann-Pick disease Type C. This decision was made shortly after analyzing the positive…
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 entire world has been waiting for a vaccine for COVID-19 since the beginning of the pandemic in March. In response, a number of pharmaceutical companies have entered the race…
Children's Craniofacial Association (CCA) has posted tips for transitioning to adulthood for those with craniofacial differences. We've summarized some of their recommendations, but you can read the full article here.…
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