The NIH has awarded Geisinger Health System, along with the University of Washington in Seattle and Washington University in St. Louis, a $5.8 million, five-year grant to study the role of…
Rare disease drug development, which now accounts for nearly one-third of all drugs in active research & development worldwide, presents challenges that will accelerate the adoption of new development strategies…
Eurordis-Rare Diseases Europe Eurordis-Rare Diseases Europe is an alliance of patient organizations which works to give rare patients a voice. It also strives to spread awareness of rare diseases to…
Heather White Heather White is a 41-year-old woman who was diagnosed with common variable immune deficiency (CVID) back in 2004 after fighting frequent bouts of pneumonia. Doctors believe that she…
This is the story of a brave journalist who was determined to improve care for rare disease patients in her country, North Macedonia. North Macedonia is a developing country located…
Dinesh Khanna, a professor at the University of Michigan has worked heavily with the rare disease scleroderma. Michigan has its own Scleroderma Program, of which Khanna is the director. Through…
Happy 7-11 Day! We hope everyone's on their way to get their free slurpie today! For this week's editor's choice, we're highlighting four articles. First, we discuss kratom, the controversial…
According to a story from Real World Health Care, nonalcoholic fatty liver disease and its less common, more serious form called nonalcoholic steatohepatitis (NASH), are silent but deadly conditions that…
According to a story from Pharmafield, The UK's National Institute for Health and Care Excellence (NICE) has recently approved a first in class gene silencing therapy for coverage on the…
According to a story from drugs.com, the biopharmaceutical company Mirum Pharmaceuticals recently announced that they have begun dosing the first patient in a phase 3 clinical trial. This clinical trial…
According to a publication from Ankylosing Spondylitis News, a recent study published in BMC Musculoskeletal Disorders suggests that LINC00311, a lengthy RNA molecule, is over-expressed in individuals with ankylosing spondylitis.…
According to a publication from Hemophilia News Today, an international team of researchers with members from France and the United Kingdom recently conducted a study of over 100 hemophilia patients…
According to a publication from OncLive, the Food and Drug Administration (FDA) recently granted priority review status to a supplemental biologics license application for Zejula. Zejula (generic name niraparib) is…
As reported in Biospace, xpovio (selinexor), a drug used to treat relapsed refractory multiple myeloma, received accelerated approval by the Food and Drug Administration this July. About Multiple Myeloma Multiple…
Kratom, an herbal supplement of the caffeine family used to ward off pain for chronic pain conditions, has received large grants for further research. While many consumers claim the supplement…
According to a story from Scleroderma News, a recent study has identified a host of risk factors that can increase a patient's likelihood of experiencing progressive organ damage. The identification…
According to a story from Ankylosing Spondylitis News, a recent study has found that the class of drugs known as tumor necrosis factor (TNF) inhibitors may actually be safer for…
It was announced this Monday that Fulcrum Therapeutics, a Phase 1 biotech developing therapies based on gene regulation for rare diseases, announced terms for its initial public offering (IPO), aka its…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. The event will take place in Iowa City, Iowa on Saturday July 20th, 2019. Event Summary:…
According to a publication from Business Wire, the Israeli biotechnology company Minovia Therapeutics recently finished dosing the first participant in a phase I/II clinical study of the Company's experimental mitochondrial…
According to a story from Wellcome Center Mitochondrial Research, a multi-center collaborative study that involved researchers from different sites around the UK has resulted in an improved understanding of the…
According to a publication at Markets Insider, the Food and Drug Administration (FDA) recently approved Soliris (eculizumab) as a treatment for neuromyelitis optica spectrum disorder. The approval marks the first…
.jpg)
Gov.UK recently published a speech by Baroness Blackwood in which she announced that the National Health Service (NHS) is currently involved in one of the most significant transformations in social…
According to a publication from the Oklahoma Nursing Times, Kenneth Wyatt celebrated his 80th birthday this past June 4th. The happy occasion also marked almost five months exactly since Wyatt…
According to a publication from WCAX, care providers at Children's Hospital Colorado are devising new ways to make life with eosinophilic esophagitis easier. One patient, six-year-old Eliana Yelpaala, is already…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
