The survival rates for infants and children with brain and spinal cord cancer has improved significantly over the past decades. However, a study published online in early August of…
According to a story from Live Science, a species of tick that is originally found in Asia has begun to appear along the East Coast of the US. The parasite…
Calling all members of the GNE Myopathy community! The Neuromuscular Disease Foundation (NDF) is hosting its 5th Annual Symposium on GNE Myopathy this August 30-31st at the University of California, Los Angeles. For…
According to DAIC, Akcea Therapeutics Inc., has recently announced their publication of results from their study analyzing patient-to-patient connectivity towards the management of the rare disease familial chylomicronemia syndrome (FCS).…
According to a story from stuff.co.nz, a baby from Auckland has been diagnosed with two extremely rare disorders that only occur in one in every 10 million people. Her name…
A recent study into Periodic Fever, Aphtous stomatitis, Pharyngitis and Adenitis (PFAPA) syndrome investigated the impact of the condition on children and found that is was associated with a reduction…
Gemphire Therapeutics Inc. has been recommended to stop a clinical trial by the Data and Safety Monitoring Board at Emory University School of Medicine. The clinical trial, which ran into…
Let's take a moment during these dog days of summer to recognize the SMA community during SMA awareness month! Spinal muscular atrophy (SMA) is a rare genetic disorder that causes…
According to a story from Newswise, an organization called the National Comprehensive Cancer Network (NCCN) has recently released treatment guidelines for a rare type of cancer that can affect women…
According to a story from Kaiser Health News, many patients that are dealing with chronic pain feel stuck in the debate over opioids in the US. As the ongoing crisis…
At the 2018 Aplastic Anemia and MDS International Foundation’s (AAMDSIF) International Bone Marrow Failure Disease Scientific Symposium of March 2018, Dr. David Margolis from the Medical College of Wisconsin, and…
According to a source article on Business Wire that can be found here, the Committee for Orphan Medicinal Products (part of the European Medicines Agency) has recommended that the experimental…
According to a story from Market Screener, the biopharmaceutical company MediciNova, Inc., recently announced its intention to begin a Phase 2/3 clinical trial for its product MN-166. MN-166, also known…
Patient Worthy exists because there's just not enough visibility for the nearly 30 million Americans who have been diagnosed with disorders that are rare or have limited treatment options. So…
Besides the obvious need for ties within the medical, pharmaceutical, and social industries, I have always wondered internally why it is so necessary that parents, adults, children, and loved ones…
According to a story from Benzinga, the pharmaceutical company BioCryst Pharmaceuticals, Inc., recently announced that its investigational product BCX7353 has been granted Fast Track Designation from the FDA for the…
We're happy to introduce a new partner to Patient Worthy's mission of advocating for rare disease patients! The Aplastic Anemia and MDS International Foundation (AAMDS) supports, connects and educates patients, caregivers…
A study is planned to investigate the effects of the experimental drug PTC596 in children with recently diagnosed diffuse intrinsic pontine glioma and high-grade glioma. The study is made up…
July 13th was Gastrointestinal Stromal Tumors Awareness Day! And that "13" has extra relevance. 5,000 Americans are diagnosed with Gastrointestinal Stromal Tumors (GIST) each year. That’s 13 people diagnosed each…
Who couldn’t use a little extra money now and again? Better still, what if earning that money was as easy as a mouth swab? According to a report on Journal…
According to a story from the FH Foundation, the non-profit organization has released a vital consensus statement regarding the diagnosis of familial hypercholesterolemia, which is the most prevalent genetic cause…
Patient Worthy’s partner the MDS Foundation will be hosting a patient forum on August 11th, 2018 from 9:30am-2:00pm "Whether you are a newly diagnosed patient, a long-term survivor, or a…
According to a story from The Sun, Alex was just two months old when he first experienced symptoms of xeroderma pigmentosum. His parents took him out to the park and…
Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…
Meet Ruby, a beautiful 3 year old who was diagnosed with hypomyelination with atrophy of basal ganglia and cerebellum, or H-ABC, shortly after her birth. H-ABC is a type of leukodystrophy…
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