Usher syndrome is a rare genetic disorder that is characterized by deafness and often paired with retinitis pigmentosa (RP), an inherited disease which causes retinal degeneration and vision loss. There…
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…
Dr. Muna Naash received her PhD from Baylor College of Medicine, where she studied retinal cell and molecular biology. Throughout her career, Dr. Naash has become known for her work…
Happy Monday! This week, we have details on a method for detecting Alzheimer's 17 years in advance, the story of a teen living with Usher syndrome, and a possible breakthrough…
According to a story from klfy.com, 15 year old Mia Braseaux may seem like a normal teenager; she plays sports, goes to school, and socializes with friends. However, Mia was…
Inherited retinal diseases (IRDs) are a group of genetic ocular disorders characterized by vision loss or blindness. A few examples are retinitis pigments (RP) and Usher syndrome. There has been…
When Krista Webb was just two years old, doctors determined that she had some level of hearing loss. Her family was surprised about this; nobody else in her family, and…
ProQR Therapeutics is working to develop unique therapeutic solutions for genetic eye diseases. Currently, the company is evaluating its therapeutic candidate ultevursen within the Phase 2/3 Sirius and Celeste clinical trials; you can read…
Rare Disease Week 2022 Rare Disease Week is the week that leads up to Rare Disease Day, which is recognized on the last day of February. This year, we are…
If you would like to read the first part of this interview, please take a look at Part 1 here. In the first part of the interview, I spoke with…
Clinical trials, drug development, and medical research have the potential to change the therapeutic landscape for patients with a wide variety of conditions. At ProQR, founder and CEO Daniel de…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Vision loss is a symptom associated with a number of disorders, one of which is Usher syndrome. Medical professionals have spent years trying to fully comprehend the underlying mechanisms of…
According to a news release from RNA therapeutics company ProQR Therapeutics N.V. ("ProQR"), the first patients have been dosed in two Phase 2/3 clinical trials evaluating QR-421a for USH2a-mediated retinitis…
At Patient Worthy, we love to see patient advocates and the impact that these advocates have on their communities. For example, brothers Ethan and Gavin Morrobel have worked tirelessly as…
Becca Meyers, a 26-year-old swimmer who was born deaf, has just been told she can't bring her care assistant to the Tokyo Games. As a result, she has had to…
The Virtual International Scientific Research Conference May 13-14, 2021 "Therapeutic Strategies for Large Protein Coding Genes in Usher Syndrome" This meeting will serve as a convergence of scientists and investigators…
The Usher 1F Collaborative has recently announced the opening of registration for its scientific conference titled "Therapeutic Strategies for Large Protein Coding Genes in Usher Syndrome." This event will be…
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…
Genetic Counselors are an incredible resource – they can help assess your risk of developing a condition, the risk of your children inheriting a condition, and what potential treatment or…
In a story for Drug Development & Delivery, the CEO of the biotechnology company ProQR, Daniel de Boer, recently sat down for an interview. In this interview, Mr. de Boer…
According to a story from newton.wickedlocal.com, the first time that mother Melissa Chaikof realized that something was wrong with her oldest doctor was many years ago. When a nearby door…
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After twenty-two years physician Huang Chun-jung finally learned why his vision and hearing have fallen to thirty percent of normalcy, thanks to research conducted by the National Health Research Institutes…
The Importance of Braille For any child with a rare disease that leads to blindness or visual impairment such as Stargardt disease, Usher Syndrome, Alström syndrome, or Retinal Blindness life…
According to a story from globenewswire.com, ProQR Therapeutics N.V. recently announced that it has begun dosing patients in its Phase 1/2 clinical trial. This trial is testing the company's investigational…
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