POTS and Vasovagal Syncope: A Patient Story
In January-March of 2015, I was the healthiest I’d ever been... or so I thought. I worked out, drank plenty of water, had a great lifestyle and meal plan. In…
Free MDS Event in Ohio!
Our partner, the MDS Foundation, is sponsoring another free event for patients and caregivers. Event Summary: Whether you are a newly diagnosed patient, a long-term survivor, or a caregiver this…
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Treatment for Childhood and Spinal Cord Cancer Linked to Lower Levels of Independence for Survivors
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…
<a href="https://pixabay.com/users/danielbahrmann/">danielbahrmann</a> / Pixabay
A New Invasive Species of Tick Has Been Found in the US. Here’s What You Should Know
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…
Register Now to Attend the Symposium on GNE Myopathy for Free!
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…
Results from First Study on Patient-To-Patient Connectivity in Those with FCS
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).…
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At Just Weeks Old, a Baby From Auckland is Diagnosed With Aicardi Syndrome and Opsoclonus Myoclonus Syndrome
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 href="https://pixabay.com/users/dagon_/">dagon_</a> / Pixabay
Study Finds Children with PFAPA Syndrome Have a Reduced Health-Related Quality of Life
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…
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A Clinical Trial of Gemcabene in Paediatric Patients with Non-Alcoholic Fatty Liver Disease Has Been Stopped Following Safety Concerns
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…
<a href="https://pixabay.com/users/Yukihide/">Yukihide</a> / Pixabay
August is Spinal Muscular Atrophy Awareness Month!
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…
<a href="https://pixabay.com/users/nguyentungson/">nguyentungson</a> / Pixabay
Guidelines for Rare Pregnancy-Related Cancers Released for the First Time
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…
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Patients With Long Term Pain Feel Stuck in the Middle of the Opioid Debate
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…
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Aplastic Anemia and MDS International Foundation: 2018 Scientific Symposium
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…
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OMS721 is Expected to Receive Orphan Drug Status for Use in Hematopoietic Stem Cell Transplantation
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…
<a href="https://pixabay.com/users/Clker-Free-Vector-Images/">Clker-Free-Vector-Images</a> / Pixabay
Clinical Trial for Potential Degenerative Cervical Myelopathy Drug Announced
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…
Watch: HelloFresh Ad Centers on Woman With Multiple Sclerosis
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 optionss. So…
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A Conversation on Rare Disease Community
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…
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Experimental Treatment for Hereditary Angioedema Gets Fast Track Designation
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…
Welcome AAMDS, a New Patient Worthy Advocacy Partner!
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…
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A Planned Study Will Investigate the Effects of an Investigational Drug in Children with Glioma
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…
#ICYMI: Join the Rare 13 Campaign for Gastrointestinal Stromal Tumors Awareness
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…
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Is There a Paycheck in Your DNA? The Answer Might Surprise You
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…
<a href="https://pixabay.com/users/PublicDomainPictures/">PublicDomainPictures</a> / Pixabay
New Statement Released by the FH Foundation on Genetic Testing for Familial Hypercholesterolemia
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…
Free MDS Event in Minnesota this August!
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…
<a href="https://pixabay.com/users/josealbafotos/">josealbafotos</a> / Pixabay
The Prosthetic Mask Protects Xeroderma Pigmentosum Patient in the Sun
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…