For the last eight years, the National Organization for Rare Disorders (NORD) has developed a State Report Card; the goal of this project is to evaluate the effectiveness of…
In a word, nothing. In two words, don’t panic. In two other words that are the same but are more positive and action oriented…be calm. Let me explain…when we experience…
Lia Porcano was born in March 2018—and her parents Rosalyn and Justin could not have been more excited. But when Lia’s newborn screening test came back abnormal, the parents’ concern…
There are many rare or congenital diseases that, without early detection and treatment, can cause serious health issues and complications. For example, early treatment of homocystinuria (HCU) could delay or…
Eight years: that’s how long it has been since Moxie Garrison was diagnosed with Marfan syndrome. Now nine years old, the spunky and resilient third grader approaches every challenge head-on.…
In a news release from commercial-stage biopharmaceutical company Calliditas Therapeutics AB CALT ("Calliditas"), the company shared positive topline results from the Phase 3 NefIgArd study. Within the study, researchers…
Reducing the spread of viral illnesses is of crucial importance to countries around the world. One such viral illness is Zika virus, a mosquito-borne illness that can be extremely…
According to a story on pharmaphorum.com, the UK's National Institute for Health and Care Excellence (NICE) has given a positive opinion for the drug vutrisiran (marketed as Amvuttra) as a…
Amber Freed has long been a tireless advocate for the rare disease community. After two years of IVF, she became pregnant with her two children: Maxwell and Riley. About four…
Cyndi Strzynski was always a bubbly, active little girl. Even though she has been facing an immense challenge, she still tries to keep a smile on her face at all…
In 2020, Lily Tyson, aged 12 at the time, was diagnosed with a rare chromosomal disorder called Turner syndrome. More recently, Lily began experiencing various eye issues: redness, pain, blurred…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Within the last year, there have been spates of cholera outbreaks in multiple countries across the globe; over the past few months, these outbreaks have touched at least 22 different…
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…
The Orphan Drug Act was passed with a goal in mind: to incentivize the development of therapeutics for orphan drugs. These drugs are used for patients with rare conditions,…
Shifts in treatment paradigms can improve the lives of countless patients and contribute to better overall outcomes. In a news release from February 23, 2023, Sanofi and international biopharmaceutical…
When Cheryl Navar decided to have a child, in-vitro fertilization (IVF) seemed like the right move. Navar knew that she had a cancer gene and didn’t want to transfer…
I have lived with dystonia (a neurological movement disorder) and chronic pain for over 20 years. The very active life prior to dystonia is night and day compared to the forced sedentary…
According to a story from World Pharma Today, Andelyn Biosciences, Inc., which is a gene therapy Contract Development and Manufacturing Organization (CDMO), has recently struck a partnership with Odylia Therapeutics,…
Together, Sarah Hutchesson-Webb and her fiancé Jake Powell have built a beautiful life together. They welcomed sons Hunter (5) and Aston (2) into the world—and wanted to round out their…
In August 2021, the U.S. Food and Drug Administration (FDA) approved Opdivo (nivolumab) for the adjuvant treatment of a rare form of cancer called urothelial carcinoma. More particularly, the drug…
If there’s one way that 11-year-old Cristian Muñoz would describe himself, it would be an athlete. Cristian has always been drawn to playing sports: the rigor, the competition, the connection…
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…
According to a story from VICE News, Congressman George Santos was recently elected to Congress in the 2022 mid-term elections. Since then, his past has been placed under scrutiny, and…
Written by Meghan Bayer Stiff Person Syndrome Spectrum Disorder (SPSD) is an acquired, progressive, neurological spectrum disorder with features of an autoimmune disease that is believed to affect one to…
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