Emily Ventura has never met in-person another person living with progressive familial intrahepatic cholestasis (PFIC), her daughter’s life-threatening ultra-rare genetic disease. That will change on June 21st when Emily and…
According to a story from PR Newswire, the Novartis company AveXis recently announced that the US Food and Drug Administration (FDA) has approved Zolgensma, making it the first ever gene…
Patient Worthy's partner the MDS Foundation is hosting a MDS Patient & Family/Caregiver Forum Baltimore, Maryland in conjunction with the University of Maryland Marlene and Stewart Greenebaum Cancer Center. This…
Dutch Bros Coffee is hosting their 13th annual fundraiser for amyotrophic lateral sclerosis (ALS) this Friday, May 10th, 2019. It's called "Drink One for Dane" in honor of Dane Boersma,…
Upcoming Conference The International Society for Mannosidosis & Related Diseases (ISMRD) serves as an international patient advocate for those living with glycoprotein storage diseases. These include alpha-mannosidosis, fucosidosis, mucolipidosis type…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. When: Saturday, April 27, 2019 Where: West Penn Hospital 4800 Friendship Avenue Pittsburgh, PA 15224 Wintergarden Conference Center – First…
According to a story from Gene42, a new rare disease platform called Genomics4RD has been launched in Canada. This is the first national research platform dedicated to rare disease to…
Second Annual International Congress on Advanced Treatments in Rare Diseases The first International Congress on Advanced Treatments in Rare Diseases, otherwise known as RARE2018, was held in London, England. Now,…
According to a story from Pharmacy Times, the US Food and Drug Administration (FDA) have recently approved the first ever therapy for the rare disorder acquired thrombotic thrombocytopenic purpura (aTTP),…
A rarely addressed subject that comes up in discussions about people with disabilities is the subject of romantic relationships, intimacy, and parenting. More than likely, a lot of day to…
Check out part one of Seth's story here. Seth had set a google alert for his hometown, Natick, to stay up to date in his home's local news. One day,…
This wonderful story was brought to Patient Worthy by our partners, the Cystinosis Research Network (CRN). The season of giving started off in an unconventional setting for the Moore Family. Chandler…
When Seth was twelve years old, he and the rest of his family began to realize something was wrong with his mother. Seth can remember conversing with her pleasantly, and…
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…
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