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…
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…
As many people within the rare disease or chronic illness community know, having support is crucial and important. Being able to find support means that you find others with…
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…
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…
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…
As originally reported in Live 99.9; when Melissa and Chris Courson found out the baby they were expecting in a few months would be born with the rare thantophoric dysplasia,…
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…
The 2019 LPA 62nd Annual National Conference San Francisco, California The Little People of America's annual conference is the ultimate experience for people affected by dwarfism and diastrophic dysplasia. This…
According to a story from abc13.com, Washington state appears to be poised to become the third US state to ban dwarf tossing, a humiliating public spectacle used as entertainment in…
A 64 year old woman recently passed away on May 11, 2018. Ellen Maud Bennett had been feeling unwell for years, and when seeking out medical treatment, her doctors did…
LPA National Conference Orlando 2018 This event will be a great opportunity for those affected by dwarfism and diastrophic dysplasia to build community and have fun with one another. This…
Mallory and Dave Holmes were excited. Like many couples, they were eagerly awaiting the birth of their first child. After 20 weeks of pregnancy, however, the couple received news that…
3D printing has officially hit the medical field and it's already making noise, reports The Jerusalem Post. Recently, 3D printing was used in Israel for orthopedic surgery and found great success.…
Warwick Davis is a big name within the Star Wars community. Those that follow the movie universe religiously will know him for his role as Wicket the Ewok in Return…
Happy Pre-Halloween Patient Worthians! October is Dwarfism Awareness Month. Not only that, MDS World Awareness Day was just two days ago! Get the deets below. We also have a story…
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