Rare disease therapies have an exorbitant cost. Fewer patients means higher costs in order to support the development of treatments. In the United States, 966 billion was spent in 2019…
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Rare diseases are much more common than people think. The word "rare" is off-putting. But the word only pertains to each individual rare condition. Collectively, rare diseases are quite common.…
Rare Disease Day is February 28th. This year, it caused a lot of reflection among healthcare professionals and families impacted by rare conditions. How has the pandemic shifted progress in…
Dicaprio Bootle is on a mission: to become a star athlete in the NFL. However, he's preparing to give back along the way to his goal. Dicaprio is fundraising for…
According to a recent article in the Seattle Cancer Care Alliance, the FDA’s recent approval of Axi-cel (Yescarta) will improve the chances of survival for relapsed or refractory follicular lymphoma.…
March is recognized each year as Bleeding Disorders Awareness Month, an event meant to help spread awareness about bleeding disorders among the general public and in the medical community. Bleeding…
At first, everything in Amberlyn Smith's pregnancy seemed to be going smoothly. When she was just over 29 weeks pregnant, her screening tests appeared to be normal. However, at…
Congress has recently introduced a new bill that would have a significant impact on the rare disease community if it passes. The legislation, called the Speeding Therapy Access Today (STAT)…
According to a story from bloomberg.com, the biopharmaceutical company Rallybio has announced two development programs for therapeutics intended to treat rare diseases. The two programs consist of RLBY212, which is…
Suddenly Sleepy Saturday, a day dedicated to spreading awareness about narcolepsy, is being held on March 13, 2021. The Narcolepsy Network is looking for volunteers to send a proclamation request…
March is recognized as Myeloma Action Month, a time dedicated to spreading awareness among the general public and the medical community about multiple myeloma, a rare hematological cancer. This event…
According to a story from Market Watch, the gene therapy company AVROBIO, Inc. has recently announced that its investigational gene therapy candidate AVR-RD-04 has earned Orphan Drug designation from the…
Mike Jackson's young daughter Evelyn was diagnosed with Batten disease after a neurologist suggested a genetic test which the family could take for free. John Barclay's daughter River passed away…
A recent study published in Cell Reports has uncovered a mechanism where germs living within the lungs of cystic fibrosis (CF) patients produce a type of carbohydrate slime that minimizes the efficacy…
James Dahlman, a scientist and professor at both Emory University and the Georgia Institute of Technology, has partnered with the CMT Research Foundation. The aim of this partnership is to…
A new study published in JAMA Oncology has found that of all patients who have had cancer spread to their bones and started treatment, 2,8% have developed osteonecrosis within their jaw, also…
A recent study has illuminated the need for further research into how different forms of exercise may provide certain benefits for patients living with Charcot-Marie-Tooth disease (CMT). This study was…
Sarcoma Sarcoma is a cancer that accounts for 1% of all adult cancers. It can affect individuals of any age and can impact any part of the body. It grows…
The FDA has just announced their approval of Evkeeza as a therapy for those living with homozygous familial hypercholesterolemia. The therapy is owned by Regeneron Pharmaceuticals. It was evaluated with…
In a recent study, researchers have uncovered a second gene located within the mitochondria that is associated with Charcot-Marie-Tooth Disease (CMT). This study was recently published in Annals of Neurology. CMT…
Lysogene has just announced that the FDA has approved their Investigational New Drug Application (IND) for a therapy for GM1 gangliosidosis, a rare pediatric condition. The investigational treatment is a…
According to a story from azfamily.com, Reyna Felix, age 28, was at work as a 911 dispatcher when her coworkers said that she started behaving strangely. Around midnight, she fell…
According to a story from ktvl.com, seven year old Emma Suetta, of Etna, California lives with a rare disease called cystinosis, and she is doing her part to help find…
The last day in the month of February is recognized each year around the world as Rare Disease Day. The day serves primarily as a moment of spreading awareness about…
According to a story from wavy.com, Cherie Reenee Brown was a prominent political organizer and activist in the city of Portsmouth, VA. But on New Years Day 2021 she passed…
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