Continued From Part One Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering.…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose of this status, “rare” conditions…
Kyleigh Kista, a 3 year old from Benonin South Africa needs heart surgery within the next 3 months to save her life. Kyleigh was diagnosed with tetralogy of Fallot with…
Pursuing an education can be difficult enough without any other extraneous factors. But what if you were also battling frequent infections, gastrointestinal problems, and recurrent hospitalizations? For Evan Brandon,…
The Myrovlytis Trust, a UK Charity focused on promoting research and advancing education for rare diseases, and Patient Worthy are now partners! This organization has focused primarily on Birt-Hogg-Dubé syndrome…
A new study has demonstrated that plasma desmosine could be an acute aortic syndrome biomarker. This also means it could be a potential diagnostic tool. The findings from this study…
One 66-year-old patient who lives in Poughkeepsie, N.Y. received the same diagnosis from three doctors. Each doctor suggested that his symptoms pointed to temporomandibular (TMJ) joint pain. The patient and…
After two years of in vitro fertilization treatments, Amber Freed, Age 38, and her husband Mark Freed of Denver, Colorado welcomed their twin babies Riley and Maxwell into the world.…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
According to a recent article, a new study from Boston Children’s Hospital shows that a first-of-its-kind gene therapy technique has succeeded in reversing severe hearing loss from a mutation to…
A recent article published in Vascular News highlights the importance of plasma desmosine, an elastin component, that provides elasticity for tissue. A breakdown of elastin leads to aortic dilatation and…
January is Cervical Health Awareness Month! So, why not use the momentum and goal-making spirit of New Year's Resolutions to make sure you get screened for cervical cancer and schedule…
January is not a surprising month. It takes people a while to remember to write the correct year, your Facebook feed is full of people going hard at the gym,…
According to a December 16, 2021 news release from biopharmaceutical company Applied Therapeutics, Inc., the company has initiated a registrational Phase 2/3 INSPIRE clinical trial to evaluate AT-007 for patients…
Thinking about the year-end holidays brings up a kaleidoscope of images, and an array of emotions for me. And everyone else too, I imagine. There are the vivid memories of…
In May 2016, I was diagnosed with a rare genetic neurological disease called adrenoleukodystrophy (ALD). Sadly, it's incurable and there's no medication to ease my condition. Since then, my life…
What does it mean to you to be living with a rare disease or chronic illness at this time of year? Over the years, since I was diagnosed I've never…
According to a recent article, professional athletes have been forced into early retirement due to arrhythmias. Arrhythmias, which are potentially fatal, affect those who exercise too much at a higher…
On December 9, 2021, the National Organization for Rare Disorders (NORD) hosted a webinar program titled "Health Equity and Rare Disorders." This webinar was the first of a three-part series…
In 2016, Marley Berthold was born. But doctors, and her parents, noticed some concerning signs. Marley's silver hair had fallen out in clumps; she had macrocephaly (an abnormally large head)…
Nancy Yu co-founded AllStripes with a vision in mind: to unlock more treatment options for those with rare diseases. In Part 1 of our interview, Patient Worthy sat down with…
For as long as she can remember, Nancy Yu has been entranced by the intersection of science and technology. In fact, as she describes it, she imagines a world in…
A recent article emphasized the importance of being educated about our eyes. As one of the most essential organs in the body, it’s important to know some key facts. Eye…
According to a news release from the EveryLife Foundation for Rare Diseases, North Carolina became the 4th state in the nation to sign newborn screening legislation in 2021 (following Arizona,…
On November 18, 2021, the Rare Disease Legislative Advocates (RDLA) held their monthly webinar. These webinars provide the rare disease community with updates regarding relevant legislation. As the year begins…
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