The warm weather is finally here (for most of us), which means it's almost time for fun, outdoor activities! We're not the only ones who are excited to spend time…
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…
True was diagnosed with congenital amegakaryocytic thrombocytopenia (CAMT) when she was just six months old. Now she desperately needs a bone marrow transplant to survive. Her mother, Anessa Haden, is…
Over the past 14 or so months, COVID-19 has dominated the headlines. It has reshaped the way that we work, attend school, build relationships with others, and receive medical treatment.…
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A decade ago, Rob Long's dreams were centered solely on football. He was looking forward to the NFL draft after a successful college career at Syracuse University, but unfortunately, a…
According to a story from PR Newswire, Solve M.E. is gathering virtually on Capitol Hill for its 5th Annual Advocacy Week, starting April 19th. A record number of people are…
Nash Hensley is six years old, but in his short life he's already faced many challenges. He was diagnosed at birth with not one, but two rare diseases. First, Nash…
Did you know that there are over 80 chemicals, called cannabinoids, that can be sourced from the Cannabis sativa plant (marijuana or hemp)? Now, Europe is harnessing this for…
According to a story from dailymail.co.uk, 59 year old Marcia Ferguson-Roa recently died only a few weeks after unusual symptoms, such as fatigue and ulcers on her head, began to…
A recent study has found that thrombosis may be the first manifestation of granulomatosis with polyangiitis disease in children following the very first report of its presentation in a 13…
The 2021 virtual American Academy of Neurology (AAN) Annual Meeting is taking place from April 17 through April 22, 2021. During the conference, experts and researchers are offering a…
A recent study has found that aerobic and strength training exercise programs can have a long term effect on physical functioning for patients with pulmonary hypertension (PH). PH is a…
According to News Medical, researchers from the University of Cincinnati presented findings on a potentially new and effective treatment option for Pompe disease at the virtual American Academy of Neurology…
In a press release, the independent specialty pharmacy Biologics by McKesson recently announced that it has been selected to the be the sole provider of fosdenopterin (marketed as NULIBRY) by…
The Johnson & Johnson (J&J) vaccine was welcomed as the third alternative to Moderna and Pfizer vaccines but with even more perks. It was a one-time shot that used “viral…
FibroGen has just announced that their investigative therapy pamrevlumab, for Duchenne muscular dystrophy (DMD), has received Rare Pediatric Disease designation from the FDA. The treatment already was given Fast Track…
In a recent press release, RNA-targeted therapeutics company Ionis Pharmaceuticals, Inc. ("Ionis") shared the initiation of a Phase 2/3 clinical trial evaluating ION373 for patients with Alexander disease (AxD). Currently,…
The International Fibrodysplasia Ossificans Progressiva Association (IFOPA) is gearing up to recognize International FOP Awareness Day this year on April 23, 2021. The event is meant to help spread awareness…
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…
In a previous article, I talked about the importance of patient-led advocacy groups and how they can positively impact how a rare diagnosis is received and understood by patients. Do…
According to a recent news release, the FDA cleared an Investigational New Drug (IND) application for VIP152, a potential therapeutic option for patients with Richter syndrome and relapsed/refractory chronic…
Alarming details of Katy Grainger’s sepsis infection were covered in the April 2021 issue of Women’s Magazine. The interview took place over two years after Katy lost both feet and…
According to HCP Live, a new study may have discovered a connection between severe pediatric atopic dermatitis and learning disabilities. Researchers, spearheaded by Dr. Joy Wan, MD, MSCE from the…
Scholarships are being provided to rare disease patients, thanks to the EveryLife Foundation for Rare Diseases. The scholarships are each worth $5,000 and are intended for those seeking an education…
According to a story from globenewswire.com, the clinical company Prothena Corporation plc recently presented encouraging results from its phase 1 clinical trial. This trial was evaluating its investigational product candidate…
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