Julia Lefelar, a resident of Gaithersburg, MD, was telling a friend about her years of seeing different doctors trying to find the cause of her illness. After a while, her…
The Critical Path Institute (C-Path), formed in 2005, is a nonprofit, independent (public/private) partnership. The Institute evolved from the FDA’s 2004 White Paper known as the Critical Path Report and…
On January 20, 2022, the Rare Disease Legislative Advocates (RDLA) hosted its first monthly webinar for 2022. In this month's program, the speakers provided an overview of the progress that…
According to a recent article, researchers were able to produce the first images of an enzyme that may figure out the unknown cause of a rare group of hereditary neurodegenerative…
This week the Guardian reported that a pig’s heart, which was genetically modified, had been transplanted into a patient by doctors who were trying to save his life. The patient was…
According to a recent article, Castle Creek Inc. has acquired Novavita Thera Inc. in an effort to expand its research and development efforts. Castle Creek Inc. Castle Creek Biosciences Inc.…
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…
Newborn screening (NBS) is a public health service within the United States through which babies are screened for medical conditions that otherwise wouldn't be apparent at birth. Many of the…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
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…
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