CFRI's third annual Embrace Retreat is happening May 5th - May 7th! The Embrace Retreat is specifically geared toward mothers of children and adults battling cystic fibrosis. Just in time…
Happy Friday Patient Worthians! April is Sjogren's Awareness Month! There was also a whole day dedicated to Bohring-Opitz Syndrome. The MDS Foundation has another upcoming event as well. Lastly, last month…
A clinical trial is up for those suffering from Duchenne Muscular Dystrophy. An oral tablet called Idebenone will be compared to a placebo, to see if it is safe and…
Welcome back Patient Worthians! This week we have some exciting news for the MS community. Additionally, we have some great tips on how to build your CF dream team. Also,…
Vertex Pharmaceuticals announced that two phase 3 studies involving a combination of tezacaftor / ivacaftor improved the lung function in cystic fibrosis patients that are 12 and older, with the F508del…
In two weeks, Ocrevus will be available to those suffering from both relapsing-remitting Multiple Sclerosis (MS), the most common form, and primary progressive MS, which affects about 10 to 15…
The World Alliance of Pituitary Organizations unites the pituitary patient community and as one of our partners, they sent us their latest newsletter! In it, you can read the chairperson's report,…
Happy Spring Patient Worthians! This week we have pieces from two rare women, one battling the rare disease pemphigus vulgaris, part of the pemphigus and pemphigoid family. The other is…
This week is a small win for adrenoleukodystrophy (ALD) awareness and for these mini-bikers. Kevin Bean're and Tom Ford are mini-biking across the country, from Florida to California, to raise awareness…
Iva Rauh is a Pemphigus Vulgaris patient and advocate living in Maryland. Originally from Eastern Europe where the gene for this disease is prevalent, this is her plea to the…
Welcome back to Editor's Choice! This week we have an interesting take on Parkinson's treatment. We also have an empowering article from one of PW's rare disease contributors. We are all feeling the…
The Rare Impact Awards for 2017 will be held this year, on May 18th, at 6:30 pm in Washington, DC. The Rare Impact Awards is the National Organization for Rare Disorders (NORDS)…
After lobbying during Rare Disease Week on Capitol Hill a couple of weeks ago, I was convinced we were pretty successful with our congresspersons in showing them how vital NIH…
I recently covered an author who had some solid advice for drug makers in regards to the rare disease pricing problem. The following drug maker may or may not have…
Imagine, three to 10 months with your new born baby in a Neonatal Intensive Care Unit (NICU) born with a potentially mortal disease or defect. You’ve battled through surgeries, recoveries,…
Friedreich's ataxia (FA) is a rare and painful disease. With few treatments available to treat the genetic mutation, most therapy is aimed at controlling symptoms. Clearly, these short-term band-aids aren't…
Chucky Bartolo of Lovin' Malta introduced Jake Vella to the world yesterday. Jake suffers from the rare disease ROHHAD. While he was a regular kid for the first five years of his…
We are huge proponents of the 21st Century Cures Act, as it does so much for the rare community. However, there has been an unintended consequence of it's 2016 passing…
Irrelevant, insignificant, ignored, un-empowered, but worst of all INVISIBLE. Let me let you in on a big secret. Those of us who are fighting everyday with the simplest of tasks,…
Sharks have a fascinating immune system (and as far as we know, they don't develop idiopathic pulmonary fibrosis (IPF). They can repair wounds and injuries quickly. Researchers even believe that…
Patient Worthy's very own Angie Randall, a mom, wife and friend with multiple sclerosis is walking to end MS. On Sunday, April 30th, 2017 at 10:45 am, at Soldier Field…
Genetic testing for rare diseases such as spinal muscular atrophy (SMA) before you conceive can be an important step in starting or building a family. It can be vital to…
Researchers at Massachusetts General Hospital (MGH) have found that mice with MC1R, the gene that is responsible for red hair, are more susceptible to toxins that are damaging to neurons…
During Rare Disease Week on Capital Hill, on behalf of Lyme and dysautonomia, in addition to ALL rare disease, I lobbied my congressman to support the newly introduced (kind of) OPEN…
We are back from Rare Disease Week in Washington, DC! And do we have some highlights for you below?! Rare Disease Day at NIH was not only informative, but encouraging.…
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