Passage Bio has been granted the Rare Pediatric Disease designation for their GM1 gangliosidosis treatment, PBGM01. This designation means that Passage will receive a priority review voucher, and it is…
After six decades of attempting to solve chronic organ rejection, researchers at Houston’s Methodist Hospital and Pittsburgh University have met the challenge. According to an article in Medical Xpress,…
By Jodee Redmond from In The Cloud Copy The results of a new study using laboratory rats to model cerebral palsy with spasticity (muscle stiffness) accompanied by dystonia (involuntary muscle…
Genetic research is a burgeoning and exciting field. With the discovery of new genetic mutations and variants comes a deeper understanding of rare diseases and their subtypes. According to Cancer…
As reported in Multiple Sclerosis News Today, Jessie Ace tells the story of receiving her diagnosis with multiple sclerosis at 22. She wrote, "I hate needles." She ranks being told…
Christopher Cowan and his lab at the Medical University of South Carolina recently conducted a study in mice in order to understand how defective MEF2C genes impact brain development. A…
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NORD has received thousands of phone calls and emails each week from concerned rare disease patients. They are asked for advice and how to decipher the myriad of contradictory information…
By Natalie Homan from In The Cloud Copy At 41, Tiffany Wedekind, of Columbus, Ohio, has lived much longer than her doctors could have hoped when she was diagnosed with…
Patient Worthy is working with a partner agency who is looking to engage with patients living with Shingles. If chosen, participants will have the chance to take part in a…
By Natalie Homan from In The Cloud Copy The new reality of the COVID-19 pandemic means that workplaces are changing very quickly. This requires every employee to adapt, and neurodiverse…
In a story from MedCity News, while some studies grind to a halt with coronavirus restrictions, some of the medical world is using the new limitations to formulate creative solutions…
By Natalie Homan from In The Cloud Copy In the United States, a disease is considered “rare” if it affects less than 200,000 Americans at any given time. Diseases can…
By Danielle Bradshaw from In The Cloud Copy It has been shown in a recently published trial that satralizumab could potentially reduce the risk of neuromyelitis optica spectrum disorder (NMOSD)…
By Jodee Redmond from In The Cloud Copy Dr. Kristina Simonyan has been studying patients with laryngeal dystonia. Through the scope of her work, she has heard numerous anecdotal accounts…
As reported in Vox, Amber Freed set into to motion when she learned her son Maxwell's genetic disorder made him just the 34th in the world to be diagnosed with…
Patient Worthy is honored to present Dr. Eunice Wang, who has herewith responded to questions from our readers about the effect of the COVID-19 virus on their cancer treatment. Dr.…
As a child, my favorite fairy tale was about a princess who is locked away in a tower and guarded by a terrible dragon. Finally, a prince comes along, slays…
As reported in Technology Network, a group of international scientists from the VIB-UGent Center presented a new antigen immune cell. These antigen-presenting cells belong to a group of dendritic cells…
A recent Phase 4 study has shown that Opsumit can improve function of the right heart ventricle for patients with pulmonary arterial hypertension (PAH). The right ventricle pumps blood to…
From social distancing and shuttered businesses to personal health concerns, COVID-19 has changed daily life for the foreseeable future. There are nearly 4.75 million diagnoses worldwide, with 1.53 million in…
A recent article in US News and World Report gives an account of the first patient to have reprogrammed cells successfully implanted in his brain. The patient’s doctors reported that…
Biological robots, commonly known as biobots, mimic the actions of other organisms (e.g. jumping or swimming). Biobots are composed of artificial materials and also organic material. As published in…
Atypical hemolytic uremic syndrome (aHUS) is a rare condition which can be sparked by pregnancy, although it can also occur without it. Essentially, if you are genetically predisposed to the condition,…
We need controls! No, not cockpit controls or videogame controls, and no, we're not out of control. We need control participants for the Dupuytren Biomarker Discovery Pilot Study. Our goal is…
The UFC has some tough fighters in it. But 35-year-old Joe Ellenberger never thought that his most difficult opponent would be within himself. That is, his paroxysmal nocturnal hemoglobinuria (PNH),…
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