According to Pharmacy Times, the FDA recently granted Priority Review status to luspatercept-aamt (Reblozyl). The therapy, for which its developer has submitted a supplemental biologics license application (sBLA), is designed…
I've certainly never been a runner -- and neither was Diane Owens, at least for a while. The mother, who lives in Southbury, CT, admits that it used to be…
In a late October news release from pharmaceutical company Marinus Pharmaceuticals, Inc. ("Marinus"), the company shared that it would be collaborating with the Loulou Foundation, a private UK nonprofit dedicated…
Let us begin this article recently published in MedCity News by starting with the ending: in order to help millions of rare disease patients waiting for rare disease treatments, a…
For years, Debra Papagni Denmark worked to have New York State recognize Phelan-McDermid Syndrome after her son Drew, now 13, was diagnosed. A few years ago, Governor Andrew Cuomo heard…
According to a recent article in Yahoo News, unless treatment is discovered or a cure is found for a disorder called IRF2BPL, these young children will lose the ability to…
In August 2021 News Center Maine ran an article introducing former British army Major Chris Brannigan. Brannigan, forty-one years old, was prepared to do anything to help his daughter Hasti,…
On October 25, 2021, Rare Disease Legislative Advocates (RDLA) held a virtual briefing for the Rare Disease Congressional Caucus. The subject of this briefing was newborn screening and ongoing discussions…
According to a news release from biopharmaceutical company Mereo BioPharma Group plc ("Mereo"), the Osteogenesis Imperfecta Foundation (OIF), and the Osteogenesis Imperfecta Federation Europe (OIFE), enrollment is now complete for…
Currently, there are no known cures for CDKL5 deficiency disorder, a rare genetic disorder characterized by seizures and severe developmental delays. Treatment, rather, is symptomatic and supportive. However, a news…
When Rhonda Stevey met her grandson, Koehyn, she was immediately smitten. The grandmother, who lives in Ohio, felt like her grandson was the happiest infant she had ever met. Unfortunately,…
Paul Williamson has always been a fiercely proud father and an advocate for the rare disease community. His son James was born with Smith-Magenis syndrome (SMS), a rare genetic developmental…
With each new day and technological improvements come new discoveries about the world around us. Some of those discoveries center around medical conditions which have never been seen before. According…
According to the Daily Sabah, the Health Ministry of Turkey is taking a new approach to family planning and screening for genetic disorders. As of October 8, 2021, the Health…
The Child Growth Foundation (CGF) is a leading UK charity which supports families, adults, and children with rare growth conditions such as SHOX deficiency, Silver Russell Syndrome, and Sotos syndrome.…
Having a rare disease can feel isolating. Many people have never even heard of your condition, much less know anything about it. This is even a problem with doctors. Despite…
Medical research drives a huge amount of our understanding around rare diseases. Recently, researchers from Brown University performed a study to deepen their understanding of a rare, X-linked genetic disorder…
When you think of effective fundraisers, do you think of pickleball? Well, a Kansas community did just that. According to a video and transcript on KMBC News 9, his community…
From certain partnerships can emerge not only a better understanding of rare diseases but potential therapeutic options. As described by BioProcess International, a nonprofit organization called the Institute for Life…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat rare or chronic illnesses. For the purpose of this designation, "rare illnesses" are defined…
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…
Clinical trials can be beneficial for finding and evaluating new treatment options. Recently, biopharmaceutical company AstraZeneca shared how ALXN1840, a potential treatment for patients with Wilson disease, reached its primary…
When Karter was first born 14 months ago, his mother Breonna was overjoyed. But she quickly realized a series of upcoming obstacles when, at birth, Karter was diagnosed with Alagille…
According to a recent press release from Pfizer and biotechnology company Vivet Therapeutics, VTX-801, a gene therapy candidate for patients with Wilson disease, recently received Fast Track designation from the…
When Caren was eighteen years old her parents were, in fact, given two options. If they were unwilling to consent to a lobotomy, Caren would remain in the dark ward…
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