As originally reported in Medscape Neurology News, for the last few years, doctors and researchers have been excited by the new prospect to use an obesity drug, fenfluramine, to treat…
A recent article in BioPortfolio carried AstraZeneca’s announcement of the FDA’s approval of CALQUENCE® (acalabrutinib) for adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The approval…
As originally reported in Mirror, when Sian Hopper was in secondary school, she began to get headaches. She attributed them to stress: she had been studying for her GCSE exams,…
A recent study published in the scientific journal PLOS One has determined that patients with the rare disorder primary Sjögren's syndrome are at a greater risk of bladder problems than previously thought.…
Long QT syndrome (LQTS) is a congenital disorder that manifests as a prolonged QT interval on patient electrocardiograms. Those who are affected by LQTS are more likely to experience…
As originally reported in CheckOrphan, there's good news today for adult patients with acute hepatic porphyria, a genetic disorder which causes pain and paralysis. The Food and Drug Administration (FDA)…
CRISPR/Cas9 technology reached another milestone with encouraging results in two recent trials of CTX001. An article in Pharmaceutical Technology carried an announcement by CRISPR Therapeutics and Vertex Pharmaceuticals about…
With over 500,000 people affected by primary immunodeficiencies in the United States alone, experts are looking for ways to help doctors manage the needs of these patients. One significant…
From November 14th-16th, 2019, iPain Summit 2019 took place in Los Angeles, CA at the UCLA's Meyer and Renee Luskin Conference Center. Patient Worthy had the privilege to attend and…
Spinal muscular atrophy (SMA) is a rare disorder, but sadly, it is still the most common genetic reason that children die before the age of 2. Not only that, but…
A team of cross-disciplinary researchers at Arizona State University (ASU) is working to develop a software which patients with neurological disorders may easily use to assess their disease progression. Through…
Kristina Wolfe and Seth Rotberg first crossed paths on LinkedIn, recognizing one another as fellow patients. Kristina is passionate about public heath, health economics, and the way non-holistic approaches to…
Phase 3 Trial Results from the Phase 3 PREVENT study for neuromyelitis optica spectrum disorder (NMOSD) were extremely positive. 98% of patients in the treatment group did not experience a…
People who live with rare diseases have found that it is often difficult to get a diagnosis. Doctors look to common issues before they consider diseases that affect an…
A study conducted by researchers at UC San Diego has revealed new and helpful information about non-alcoholic steatohepatitis (NASH). This study, led by Dr. Joseph Witztum, focused on a…
Each year just under 1,500 infants are born with spina bifida, a name given to a cluster of malformations that affect the neural tube. Of those 1,500, only 43%…
According to a story from USA Today, Thanksgiving 2009 was a harrowing time for the Anderson family. About a month earlier, Darrin and Sarah Anderson's oldest son Henry had died…
Status Update: Shared Decision-Making in Hematology Sunday, December 1, 2019 Beth Faiman, CNP, PhD Certified nurse practitioner at the Cleveland Clinic in Cleveland, Ohio In this edition, ASH Clinical News…
Global Blood Therapeutics was recently granted priority review by the FDA for their new drug Oxbryta. This drug is meant to treat sickle cell disease, and it is the…
According to a story from biopharmadive.com, the field of gene therapy has gained increasing relevance throughout 2019, and 2020 will likely be a continuation of this trend. An area of…
According to a story from Myeloma Research News, the first patient has been dosed with an experimental allogeneic (donor derived) CAR-T cell therapy called UCARTCS1 as part of a phase…
Finding the correct doctor for treatment can be difficult, especially if one has a rare disease. Because few doctors specialize in rare diseases, they are often spread across the…
One thing many rare patients know is that many rare diseases do not currently have any therapies. However, that is changing. There are so many possible solutions that need coordination,…
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