A majority of rare diseases are genetic in nature, with an estimated 50-75% of these conditions manifesting in childhood. The Rare Pediatric Disease designation was established to encourage and streamline…
When it comes to rare and chronic conditions, awareness is important. Oftentimes, disease-specific information remains within the community; many others are informed about the impact of these conditions. Awareness not…
In the past, the U.S. Food and Drug Administration approved Voxzogo (vosoritide) for children with achondroplasia who were five years old or older. Voxzogo, developed by BioMarin Pharmaceutical, is…
Have you ever heard of Orphan Drug designation? This special status was created to incentivize the development of therapies for people with rare conditions (affecting fewer than 200,000 people…
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…
There are many terms that were once widely used which held offensive origins and meanings towards certain groups. Now, in a more socially conscious society, we are rightfully abandoning these…
Angela Singletary is the proud mother of three children: Aidan, DL, and Angela. One of them sticks out from the rest - literally. DL is a foot and a half…
BioMan Pharmaceuticals has just announced that vosoritide has just received a positive opinion from the Committee for Medicinal Products in Europe, which means marketing authorization is recommended. Further, it has just…
Not everyone in the little people community is advocating for the controversial drug that’s promising to increase the growth of people with achondroplasia, the most common type of dwarfism. The…
Nash Hensley is six years old, but in his short life he's already faced many challenges. He was diagnosed at birth with not one, but two rare diseases. First, Nash…
Recently, the Endocrine Society's virtual Annual Meeting, dubbed ENDO 2021, took place from March 20 - 23, 2021. During the meeting, attendees discussed the latest in endocrinology research, treatment,…
In a press release from March 3, 2021, biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") shared that it had completed full enrollment for a Phase 2 clinical trial evaluating vosoritide for…
In a recent press release, biotechnology company VISEN Pharmaceuticals ("VISEN") announced the approval of its Investigational New Drug application (IND) in China. The China Center for Drug Evaluation (CDE) accepted…
BioMarin plans to expand its clinical program for vosoritide, its treatment for achondroplasia. This expansion includes two new phase 2 trials, the first of which will evaluate vosoritide in infants…
In early November, global biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") announced the acceptance of its New Drug Application (NDA) for vosoritide. The investigational injectable treatment is designed to treat pediatric…
Last week, biotechnology company BioMarin announced that Dr. Lynda Polgreen, MD, MS would present at the American Society for Bone and Mineral Research (ASBMR) Annual 2020 Meeting. Her presentation centered…
BioMarin Pharmaceutical Inc. has recently submitted a new drug application (NDA) for their achondroplasia treatment, vosoritide. This once a day injection is for children who live with the disorder, and…
Ascendis Pharma has just announced that their investigative therapy for achondroplasia called TransCon CNP has just received Orphan Drug Designation from the European Commission. This same designation has already been…
BioMarin has recently submitted a Marketing Authorization Application to the European Medicines Agency for their achondroplasia treatment, vosoritide. As of now, the review of the application will begin in August.…
According to SeekingAlpha, biotechnology company QED Therapeutics recently announced that the first pediatric patient was dosed in the PROPEL 2 trial. The mid-stage trial, which is testing the investigational…
Recently, pharmaceutical company RIBOMIC announced that the first patient was dosed in their Phase 1 clinical trial. Ultimately, the trial is meant to determine whether RBM-007 could be an effective…
According to a story from BBC, nine year old Sam Short has been trying an experimental drug for three years that aims to improve his growth. This is because he…
About BridgeBio Pharma BridgeBio Pharmaceuticals was created in 2015 by veterans from the fields of academia and biotechnology. The company focuses on the development of new therapeutic treatments for genetic…
BioMarin has just released an update on their achondroplasia and hemophilia clinical trials. Exciting things are in the works! Achondroplasia Update Achondroplasia causes short stature. A phase 3 trial working to…
October is Achondroplasia (Dwarfism) Awareness Month! Achondroplasia is a bone growth disorder that causes dwarfism. Dwarfism is defined as a condition of short stature as an adult. People with achondroplasia are…
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