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.…
According to an article in Biospace, CureVac of Boston, MA, with headquarters in Tübingen, Germany, just announced interim results from its Phase 1 study evaluating CVnCoV, a vaccine candidate against…
CymaBay Therapeutics, a biopharmaceutical company based in Newark, CA, develops and provides access to novel therapies with a recent focus on primary biliary cholangitis (PBC). The company is currently developing…
DNA sequencing has become part of our lives - but what is it? For one thing, it is a big business. In 2003, approximately $3billion US found its way into…
As reported by the Human Rights Watch; in Mozambique, a grassroots organization to support people with albinism is using murals to spread awareness and a positive image of the disease.…
Astellas Pharma, headquartered in Northbrook, Illinois presents its Be R/Ready program to promote patient-physician communication as well as other valuable and informative resources. Their program centers around patients with relapsed…
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…
A recent article from the CCA has explained some considerations for mask types that you may not have thought about. Some of these are discussed below, but you can read…
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