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…
According to Newswise; in a new review of studies which looked at 36 studies on the COVID-19 virus found that almost one in five people with COVID-19 only show gastrointestinal symptoms,…
by Danielle Bradshaw from In The Cloud Copy Bone grafts, although very helpful, can result in hard to treat infections and typically result in the patient needing to be put…
Medical professionals are looking towards patient-reported outcomes more and more as a source for research. In fact, a study titled "Patient reported impact of Charcot-Marie-Tooth disease: protocol for a real…
According to a story from GlobeNewswire, the company BioCryst Pharmaceuticals, Inc., recently announced that its drug berotralstat (marketed as Orladeyo) will be available in the UK under the Early Access…
According to a story from UT Health San Antonio, a team of researchers have discovered in a new study the mechanism behind the activation of magnesium ions in cells. The…
As many patients with rare diseases know, it can sometimes be difficult to achieve the correct diagnosis. In fact, the average length of time it takes from symptom onset to…
A recent study published in the Journal of Translational Medicine describes an accurate and objective approach that aims to evaluate the severity of myalgic encephalomyelitis, also known as chronic fatigue syndrome. This…
Patricia Simon Patricia Simon was born with a cleft lip and palate. She is now a nurse, a public speaker, a patient advocate, and a writer. She has a great…
According to a story from Street Insider, the biopharmaceutical company Forma Therapeutics Holdings, Inc., recently announced that its investigational therapy candidate FT-4202 has earned Orphan Drug designation from the European…
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