This year, the entire Patient Worthy team traveled up to Washington, DC for Rare Disease Week 2020. This event is always a big one for the rare disease patient community.…
Susan Mann was seven years old when she was diagnosed as having Hashimoto’s thyroiditis, an autoimmune disease that affects the thyroid. According to a recent article in the online…
As originally reported in Biospace, two research networks dedicated to forming bridges between rare disease research around the world will share their niches to promote discovery for rare diseases. TriNetX…
Todd Mercer, a fifty-two year old Michigan resident and father of two, recently called into STAT’S Podcast The Readout LOUD to discuss the difficulty he has had getting enrolled…
Hope everybody is catching up after Rare Disease Week! We're so glad we got to see advocates and patients at RDW making the rare voice heard in DC last week.…
Why are rare diseases so difficult to study? Is it just their rarity? Or is it also industry challenges? Unfortunately, industry has a lot to do with it. Clinical Trial…
As originally reported in CBC News, the problem wasn't that Tom Salisbury had a cold. The problem was that it wouldn't go away. No rest and patience were enough and antibiotics…
The Boston Globe recently interviewed the principals in a case brought by the Pelletier family against the Boston Children’s Hospital. The article gives details about a family’s charge of…
The charges against Baby Jace’s parents, Crystal Bryant (age 24) and Jarvis Bryant (age 27), seemed to imply that they were guilty until two doctors took a closer look.…
Abbey Luffman was a 16-year-old from Owego, New York excited about getting her learner's permit. She was also very generous; she didn't even hesitate to check the box for organ…
Mantle cell lymphoma is a cancer that often responds well to initial treatment. Because of this, doctors often recommend intense treatment soon after diagnosis. While it is often successful, sometimes…
We're no stranger to 'Stranger Things' breakout star Gaten Matarazzo, who captured our attention playing Dustin on the hit show -- but he also captured our hearts when he became…
As originally reported in Mercer Island Reporter, Jill Hawkins's three births went smoothly. Her eldest reached expected milestones and began to walk and talk. Her daughter, born four years later,…
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Jennifer Schlegel’s life began earlier than expected with an unanticipated disease. According to an article in The Lantern, Jennifer was born prematurely and diagnosed with spastic diplegic cerebral palsy.…
The Jack McGovern Coats' Disease Foundation, a nonprofit patient organization committed to supporting critical disease research and raising disease awareness, is offering travel grants for recipients to attend the 2020…
Sedation Versus Sleep Sleep and sedation are not synonymous terms, despite the fact that they are often used interchangeably in the hospital setting. The biggest difference? Sleep is always beneficial…
Lucy Lintott is the youngest woman in Scotland to be diagnosed with motor neurone disease (MND). As it causes progressive weakness, Lucy did not think it was possible for her…
As originally reported in Pfizer, the European Commission has just approved the first treatment option ever available to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM). The medication, VYNDAQEL, taken orally once daily,…
One of the less well known forms of primary immunodeficiency is called activated PI3K delta syndrome (APDS). It is a rare disease that has only recently been identified by scientists.…
By Danielle Bradshaw from In The Cloud Copy Neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (pNFs) is a condition that many children struggle to manage due to a lack of proper…
According to a press release from Epizyme, Priority Review has been granted for tazemetostat (Tazverik), a drug used to treat relapsed or refractory follicular lymphoma (FL) for patients whose symptoms…
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According to a recent article in The Pharma Letter, the European Medicines Agency (EMA) granted PRIME (PRIority MEdicines) designation to Viralym-M based on positive results from a Phase 2 proof-of-concept…
By Jodee Redmond from In The Cloud Copy A recent study suggests that certain antibodies could be linked to specific human leukocyte antigen (HLA) genes and environmental components in scleroderma.…
By Danielle Bradshaw from In The Cloud Copy A Tufts University science team was able to identify a means to possibly reverse Friedreich’s ataxia. Friedreich’s ataxia (also known as…
By Gracie Van Brunt Hi! My name is Gracie Van Brunt, I am 25 years old and I have a rare disease. At the age of 2, I was…
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