According to a story from CBS News, Roxli Doss, an eleven year old girl, was diagnosed with a rare and dangerous brain tumor called diffuse intrinsic pontine glioma. It is…
There are only 11 people in the world diagnosed with Mitochondrial Complex 1 Deficiency. Katherine Faughn, a 7-year-old living in Kentucky is one of these 11. The disease wasn't even…
According to a story from Buzzfeed News, Stephen Hillenburg, the creator of the smash hit children's cartoon SpongeBob SquarePants has passed away at age 57. Last year, Hillenburg had revealed…
Cystic fibrosis patients in the UK are planning a protest on November 19th, 2018 with the goal of getting Orkambi, an important cystic fibrosis drug, covered on the NHS. This…
According to a recent press release, patients living with ankylosing spondylitis may soon have another treatment option. The researchers studying the new treatment, known as ixekizumab, recently posted results from…
The clinical research process is a thorough and time consuming method in which new drugs, therapies, and medical devices are rigorously tested before being made available to the public. Through…
At 14 years old, Bede Marciari has experienced and missed out on a great number of things. For many people, this is just part of growing up. In Bede’s case,…
Linda Ramsey had a son named Kevin. In the beginning everything was okay, and Kevin grew normally for a short time. After a few months, his experienced difficulty swallowing. He…
The biotechnology company Synlogic announced positive data from its most recent Phase 1/2a clinical trial of its experimental product SYNB1618. This trial did not test the therapeutic action of the…
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently approved the drug Oxervate (cenegermin). This treatment is the first FDA approved therapy for the rare…
According to a story from MD Magazine, the US Food and Drug Administration (FDA) has approved the medication migalastat, also known as Galafold, for the treatment of Fabry disease. This…
According to a story from BioSpace, an announcement from the biotechnology company Vertex Pharmaceuticals revealed that the U.S. Food and Drug Administration has approved an expanded indication for ORKAMBI, the…
PatientWorthy will be offering a pair of tickets to the Alagille Syndrome Alliance's (ALGSA) First Annual Gala. The tickets will be available for patients or caregivers in the San Diego…
According to PRNewswire, Express Scripts announced a new program to benefit people living with rare diseases on May 31st. The program, called Second Opinion is part of the company’s larger…
After years of resistance from the previously dominant Republican party, Virginia lawmakers were successfully able to pass a measure expanding Medicaid to nearly 400,000 more Virginians, according to the Washington…
According to a story from PR Newswire, the pharmaceutical company BioMarin Pharmaceutical Inc. has recently announced that the U.S. Food and Drug Administration as issued approval for their product pegvaliase-pqpz, which…
May 12th through May 16th is National Neuropathy Awareness Week! It is reported that about 1 in 15 Americans are afflicted by neuropathy - but many of them do not…
Let's get moving in May! First off, for all you pop culture/Internet meme lovers, it's that time of year... May means we're on the eve of summertime, knee-deep in springtime,…
According to a story from globenewswire.com, the pharmaceutical companies Ultragenyx and Kyowa Kirin recently issued an announcement that the U.S. FDA has granted approval to their new treatment Crysvita (burosumab-twza).…
As we close out the month of March - it's important to give a little thought to some of the lesser-known rare diseases that are often overlooked. Bleeding disorders is…
According to a story from globenewswire.com, the pharmaceutical company Amicus Therapeutics recently announced that its drug Galafold had received official approval for public use in Japan. Galafold is the first…
According to a story from raredr.com, The U.S. Food and Drug Administration (FDA) has recently approved Hizentra as a maintenance therapy for patients with chronic inflammatory demyelinating polyneuropathy. Hizentra is…
Shannon Hudson, mother from Columbus, is doing everything in her power to bring awareness to Glutaric Aciduria/Acidemia type 1, a rare disease her daughter was diagnosed with upon birth,…
Last week was World Kidney Day! And in fact, the whole month of March is kidney month - so there's no excuse to brush up on some key kidney facts…
Elizabeth Jane Allen, known best as "Betty", will be honored this week for the wonderful woman she was and the lasting impact she has made on the cystic fibrosis community, reports…
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