Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
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…
At the beginning of March 2021, gene therapy company AVROBIO, Inc. shared that its investigational gene therapy, AVR-RD-04, was given Orphan Drug designation by the European Commission (EC). This treatment…
In a joint statement, biopharmaceutical companies AstraZeneca and Merck & Co., Inc. ("Merck") shared that LYNPARZA (olaparib) achieved a superiority boundary in the Phase 3 OlympiA clinical trial. During…
According to a recent press release, neuropharmaceutical company NeurAegis Inc. shared the publication of some study data which explored calpain-2 as a potential therapeutic target for status epilepticus (SE). The…
During the virtual XVIII International Conference on Duchenne and Becker Muscular Dystrophy, specialty pharmaceutical company Italfarmaco Group ("Italfarmaco") shared updates regarding various Givinostat clinical development programs. The treatment is designed…
An estimated 1 in 10 Americans, or 1 in 20 people globally, have or will have a rare disease at some point. Rare Disease Day takes place on February 28. Ultimately,…
According to a February 25 press release, Amondys 45 (casimersen), a therapeutic option for patients with Duchenne muscular dystrophy (DMD), recently became FDA-approved. The therapy is specifically designed for patients…
On February 18, 2021, specialty pharmaceutical company Kyowa Kirin Co., Ltd. ("Kyowa Kirin") shared that a Phase 2 clinical trial evaluating KHK4083 had reached its primary endpoint. KHK4083 is an…
In some cases, elderly patients (those over 70 years old) with esophageal cancer have been told that surgery is not the best option. This stems from a belief that…
February 28th is known as Rare Disease Day, designed to raise awareness of rare diseases, patients, caregivers, and their experiences. As this day approaches, the American Kidney Fund (AKF) shared…
In 2007, biotechnology company Seagen Inc. and pharmaceutical company Astellas Pharma Inc. ("Astellas") collaborated to develop enfortumab vedotin. Now, in 2021, the pair submitted two supplemental Biologics License Applications (sBLAs)…
During clinical development, treatments are sometimes first evaluated in healthy volunteers. This is the route that Insmed Incorporated ("Insmed") took with treprostinil palmitil inhalation powder (TPIP). Now, following positive topline…
After discovering that some pancreatic cancer expresses high amounts of intratumoral interferon signaling (IFN), researchers wondered whether they could create a new plan of treatment. According to Medical XPress,…
Recently, botanical pharmaceutical company Devonian Health Group Inc. ("Devonian") has been evaluating its product thykamine for patients with mild-to-moderate atopic dermatitis in a Phase 2 clinical trial. In a…
Biotechnology company Stealth Biotherapeutics ("Stealth") has made the company's mission to develop and commercialize treatments for patients with diseases characterized by mitochondrial dysfunction. One leading treatment in the company's…
As many patients in the rare disease community know, achieving a diagnosis can be a lengthy and difficult journey. Following this journey, it can still take a while to receive…
Recently, clinical stage biopharmaceutical company Immunic, Inc. ("Immunic") has been working to develop potential treatment options for patients with primary sclerosing cholangitis (PSC), a rare condition for which no…
New research suggests that ultrasounds could be a new diagnostic tool for patients with a rare lysosomal storage disorder. According to Fabry Disease News, kidney dysfunction is characteristic in Fabry…
According to a recent press release, scientists studying the Uniformed Services University of the Health Sciences' (USU) The American Genome Center (TAGC) identified five genes which could play a role…
For nearly one year now, COVID-19 has dominated our headlines. In 2020, news emerged of SARS-CoV-2, a virus causing a global pandemic. Now, in February 2021, there are 110 million…
Just over a week ago, drug discovery and development company Rescindo Therapeutics Inc. ("Rescindo") shared that its drug candidate RSC-57 received both Orphan Drug and Rare Pediatric Disease designations. The…
In the past, researchers have questioned the exact cause of spina bifida, a neural tube defect in which the spine does not fully close around the developing spinal cord nerves.…
According to Targeted Oncology, the first patient was dosed in a Phase 1 clinical trial evaluating NBTXR3, an investigational tumor-agnostic radio enhancer, for patients with esophageal cancer. The trial will…
While gene therapy is a promising field, there are some associated risks: unwanted immune reactions, infections, or whether the therapy could lead to the development of other conditions, like cancer.…
Typically, the pathway to diagnosis for endometrial cancer can be long, uncomfortable, and invasive: transvaginal ultrasounds, biopsies. However, this may be about to change. In a recent press release, Arquer…
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