Patient Worthy is excited to announce that this Saturday a solidarity concert will be held in Noáin in favor of Dravet syndrome. United by the Dravet through Ranchera Music…
VeDA Vestibular Disorders Association (VeDA) is a nonprofit organization that was founded in 1985. Its goal is to raise awareness of vestibular conditions, improve early diagnosis, and advocate for more efficacious…
On January 21, 2021, the Rare Disease Legislative Advocates (RDLA) hosted an online webinar that focused specifically on the subject of advocating for rare disease health policy at the state…
Over the past decade, scientists have made many life-saving advances in all areas of medicine. They have created molecular agents to target neurodegenerative diseases. But they were stopped right there…
Researchers at Johns Hopkins School of Medicine noted that osteonecrosis (ON) is one of the more serious complications of systemic lupus erythematosus (SLE). According to a recent article in…
Diagnosing Rare Diseases Diagnosing rare diseases is historically difficult. Physicians are taught to think about horses instead of zebras because horses are abundantly more common. For instance, if you're tired…
Veer Basal, age 14, first started showing signs of Friedreich's ataxia when he was around ten years old. The first sign that something was wrong was when Veer began to…
Kohl's has brought a new clothing line whose target audience is rare disease patients. The new clothing includes adaptive options to allow easy access to medical ports and easy dressing.…
According to a recent article in Statnews, providers must obtain prior authorization from an insurer for several reasons; to start, the insurer often requires physicians to justify prescribing a…
As reported in MSN, Duane Zingale explained that one aspect of being born with Treacher Collins syndrome is that you’re extremely memorable. Duane was born without ears or cheekbones. He…
Note: This press release was republished with permission from the EveryLife Foundation for Rare Diseases. The Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases,…
CBC News recently interviewed Amanda, Bradley, and Brad Bright, a close-knit black family living in Nova Scotia. The Brights explain that working their way through initial diagnosis and treatment…
Parents of children who have received a diagnosis of Hutchinson-Gilford progeria syndrome (HGPS) may be relieved to learn the FDA recently approved Zokinvy capsules that were developed to lower…
According to EMJ, findings from an analysis spanning 20 years has found that weight loss surgery used to address obesity also significantly lowers a person's risk of developing pancreatic cancer.…
As reported in EMJ Reviews, the world is on alert, being told to wash their hands and keep distance amidst the COVID-19 pandemic. However, patients with inflammatory bowel disease (IBD)…
25 year old Tylia Flores wrote on her blog that as a person with cerebral palsy, there are a lot of not so thoughtful ‘compliments’ that may not be interpreted…
The initial CRISPR treatment for sickle cell disease was administered to Victoria Gray in July 2019. Victoria, a resident of Forest, Mississippi, was born with sickle cell and has…
Super T’s Mast Cell Foundation was created in 2015 by my daughter Taylor upon receiving her diagnosis of mast cell activation disorder. After traveling over twenty hours to consult with…
Research During COVID Many medical research projects have faced hurdles since the pandemic has started. Regulators have changed development timelines, and many have had to delay their clinical trials due…
The Partnership A new partnership has just been announced between Mendelian, a digital health company for rare disease, and Modality NHS partnership, a rare disease diagnosis program. Together, these organizations…
Many people have questions about the COVID-19 vaccine, a number of them pertaining to the speed at which it was developed and approved. Rare disease patients are no exceptions. Barbra…
A recent announcement by Gilead Company citing data from Kite Pharma, one of its premier companies, set out remarkable results from Kite’s Zuma-1 clinical trial. The findings confirmed that…
As reported in PR NewsWire; in an effort to help the entirety of the sickle cell disease community, Emmaus Life Sciences, Inc has announced a program that will provide their…
Eurordis The European Organization for Rare Diseases (Eurordis) is a nonprofit organization which organizes over 930 rare disease groups into one alliance. Groups have joined the network from 73 different countries.…
Looking for a feel-good story? Then look no further. In December, telecom company Cox worked to make a difference for the rare disease community. The organization headed its Elite Gamer…
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