On January 26, 2023, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
This is Part 2 of a two part story. Check out Part 1 here. The loss of his child through Edwards Syndrome helped Dr. Harsha Rajasimha understand the suffering and…
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…
PART I The baby died almost immediately after birth. Edwards Syndrome is a rare congenital disease. Only about ten percent of newborn babies survive past their first year. Babies…
When Arica Svoboda thinks of her two children, Hayes and Hendrix, she can’t help but fill up with love. She tells Patient Worthy: “My boys are special. Their smiles and…
Treatment for Alagille syndrome, a rare genetic disorder that affects multiple organ systems, are typically symptomatic. Many children born with this disorder eventually require a liver transplant; without this, Alagille…
An opinion expressed by Sheila Frame, President of Americas at Amryt Pharma, was published recently in the Pittsburgh Post-Gazette. Ms. Frame, who serves on the board of the Rare…
Right now, there are only an estimated 10 people in Ireland who have been diagnosed with a form of mucopolysaccharidosis type I (MPS I) called Hurler syndrome. This is the…
Trigeminal neuralgia is characterized by intense and debilitating chronic pain affecting one side of the face. Currently, there are a number of treatment options to reduce pain: tricyclic antidepressants, anticonvulsants,…
When she was an infant, Phoenix Radziwon was diagnosed with Down syndrome. However, her family has — over more recent years — learned that her Down syndrome diagnosis may go…
In March 2022, T. Kavinraj began experiencing a number of worrying health symptoms. He had a splitting headache and could not stop vomiting. In fact, his condition was so bad…
This year, the 13th annual Moebius Syndrome Awareness Day (MSAD) will take place on Tuesday, January 24. Moebius Syndrome Awareness Day, as the name suggests, is an event to celebrate…
New Delhi: Recent headlines in the Hindustan Times highlighted a letter written by the leader of the Bharatiya Janata Party, Varnum Gandhi, to Health Minister Mansukh Mandaviya urging him…
Rare disease stakeholders are called on to participate in the first Rare Disease Therapeutic Alliance of 2023 featuring an agenda presented by Rare Advocacy Movement experts. The agenda will…
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…
423 words 10% matched vs 524 words 5% matched The Rare Diseases International Organization reports that the groundbreaking UN Resolution on Persons Living With a Rare Disease (PLWRD), effective…
Simeon Fryer was elected captain of the Texas A&M University Corpus Christi (TAMUCC) Islanders men’s basketball team for the 2022-23 season – and it’s a job that he takes very…
Before you read on, don't forget to check out Part 1 of our interview. In Part 1, Alex discusses the multi-year diagnostic odyssey to discover that Raymond has VAMP2. Today, we talk…
Before you read on, don't forget to check out Part 1 of the story. In Part 1, Whitney talks about what CDKL5 deficiency disorder (CDD) is and the diagnostic journey for her…
On December 23, 2022, President Joe Biden officially signed the Consolidated Appropriations Act, 2023. This bill sets in stone the federal government's budget for the year, so naturally it includes…
In the United States, rare diseases are defined as those affecting fewer than 200,000 people. In the cases of ultra-rare conditions, there is often even less research, less resources, and…
A year after her daughter Havilah was diagnosed with CDKL5 deficiency disorder (CDD), Whitney Mitchell’s pediatrician looked her in the eyes and said something that Whitney still carries with her…
Do you wish to be heard? Recently PR Newswire announced that on the 28th of February 2023, NORD and the worldwide community of rare disorders will be raising awareness…
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…
Written by Lisa Ann Krutzik An inexpensive and extremely valuable gift you can give your older teen or adult child this holiday season... A letter from you. I spend a…
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