Verge Genomics, based in San Francisco is one of a series of biotechs that have entered into a new era using AI in drug discovery. The company is backed by…
Oxytocin has been deemed the "love drug" or "love hormone." This hormone, which controls aspects of behavior and reproduction, is often released when we're in love or in physical…
According to a late October 2022 news release from gene therapy company AVROBIO, the sixth patient has been dosed in a Phase 1/2 clinical study evaluating AVR-RD-04 for patients…
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…
Before you read on, don't forget to check out Part 1 of our interview with Jennifer Wallace Valdes, PT. In Part 1, Jennifer discusses her background, what Duchenne muscular dystrophy (DMD) is,…
The European Society for Gene and Cell Therapy (ESGCT) recently held its Annual Congress from October 11-14, 2022. During the ESGCT Congress, various stakeholders discussed data, trends, and new research…
The Orphan Drug Act was passed in 1983 to stimulate the development of therapeutics for rare diseases. With this came the Orphan Drug designation. This designation is granted to drugs…
Glioblastoma multiforme (GBM) is notoriously difficult-to-treat. This cancer is both aggressive and invasive, spreading “tentacles” and becoming hard to remove even with surgery. As a result, finding more effective therapeutic…
The FDA created the Rare Pediatric Disease Designation and Priority Review Voucher Program to stimulate the development of therapies intended to treat, diagnose, or prevent rare pediatric diseases. These are…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose a rare disease or condition. Rare conditions are those affecting fewer…
According to a story from prnewswire.com, the pharma company Harmony Biosciences Holdings, Inc., has recently announced the release of data from its phase 2 clinical trial. This trial is evaluating…
According to ABC News, cases of Vibrio vulnificus (V. vulnificus) infections have risen in Florida following the devastating effects of Hurricane Ian. V. vulnificus infection, which may also be referred to as vibriosis, is…
Occupational therapy for individuals with Duchenne muscular dystrophy (DMD) requires a unique and specialized approach to maximize benefit and reduce harm. It is incredibly important that those performing occupational therapy…
Did you know that November is National Family Caregivers Month? Celebrated annually, this month is designed to recognize the contributions of caregivers and honor their actions. In a recent…
At 29 years old, Morgan Nuchols is living the fullest life that she can. She loves challenging herself by doing puzzles. But her favorite activity? Making birthday and holiday cards…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A rare condition is one affecting fewer…
Glioblastoma is notoriously difficult to treat and manage. The cancer is not only aggressive and quickly spreading, but is often treatment-averse. Because of this, glioblastoma comes with a poor prognosis…
For the last few decades, there has been increased interest in understanding the effects of sleep (or lack of!) on our overall health. A recent study sought to understand the…
Before you read on, make sure to check out Part 1 of our interview with Geraldine Bliss, the President and Co-Founder of CureSHANK and mother to Charles, who has Phelan-McDermid syndrome. Today,…
Prior to her daughter Avenly’s diagnosis, Lacy Biram had never before heard of Phelan-McDermid syndrome (PMS). Currently, there are an estimated 2,200-3,000 people worldwide who have this rare condition. For…
Genetic Counselor Awareness Day is recognized on the second Thursday of November each year. In 2022, the day falls on the 10th of November. This awareness day is intended…
In 2006 twenty-nine-year-old Austinite Ben Munoz of Dallas, Texas experienced such a severe headache and dizziness that he drove to the hospital and underwent surgery several hours later. Ben was…
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…
On November 8th, 2022, CureSHANK is sponsoring an Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for Phelan-McDermid Syndrome (PMS). Co-planned by the Phelan-McDermid Syndrome Foundation, this virtual EL-PFDD meeting will: [give]…
Join thousands of people worldwide in raising awareness of mouth cancer during the month of November. It is the largest campaign in the UK in support of education and awareness…
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