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.
Just 50 years ago, receiving a diagnosis of severe aplastic anemia could be devastating. While therapeutic options have greatly improved over the past five decades, some doctors have still found…
Orphan Drug designation is typically granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases; in the United States, these are conditions affecting under 200,000 Americans. Drug…
Due to plastic pollution, microplastics can now be found -- well, nearly everywhere. Microplastics have been found on beaches and in water supplies, in our foods and in the deepest…
In a news release from January 4, 2022, biopharmaceutical company SpliSense shared that their drug candidate SPL84-23-1 for cystic fibrosis (CF) earned Orphan Drug designation in both the United States…
In Part 1 of "With Mais and Mal," Patient Worthy spoke with Mallory Cyr about microvillus inclusion disease (MID), Mallory's story, and the process of becoming an advocate. Today, we…
What does someone with a rare disease look like? With over 7,000 rare diseases in the world, affecting an estimated 300 million people, life with a rare disease (and people…
Cannabidiol (CBD) has been explored as a potential therapy for a variety of different conditions, ranging from various forms of epilepsy to dystonia to Parkinson's disease. Now, researchers are questioning…
Have you ever heard of the cytokines interleukin-6 (IL-6) or TNF? Normally, these cytokines, excreted by the immune system, may influence autoimmunity or immune responses to foreign invaders. For…
Pancreatic cancer can present numerous problems for medical professionals. To start, this cancer can be difficult to detect and diagnose in early stages. By the time pancreatic cancer progresses,…
The Phase 3 BE MOBILE 2 clinical trial sought to evaluate the safety, efficacy, and tolerability of bimekizumab for adult patients with ankylosing spondylitis (AS), an inflammatory disease affecting…
In a December 13 press release listed on the company's website, genome editing company Intellia Therapeutics, Inc. ("Intellia") shared that the first patient had been dosed in a Phase…
According to DocWireNews, on December 16, 2021, the U.S. Food and Drug Administration (FDA) granted Accelerated Approval to Tarpeyo (budesonide) delayed-release capsules. The therapy is designed to treat adult…
Clinical trials can be extremely beneficial in learning more about various diseases, as well as the safety, efficacy, and tolerability of potential treatments. According to a news release from…
On December 17, 2021, global immunology company argenx SE ("argenx") shared on its website that its therapy VYVGART (efgartigimod alfa-fcab) was approved by the FDA for the treatment of…
In a news release from mid-December 2021, rare liver disease company Albireo Pharma, Inc. ("Albireo") shared that positive topline data was available from a Phase 1 clinical trial evaluating A3907…
According to a December 16, 2021 news release from biopharmaceutical company Applied Therapeutics, Inc., the company has initiated a registrational Phase 2/3 INSPIRE clinical trial to evaluate AT-007 for patients…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. These are defined as conditions affecting under 200,000 Americans.…
According to a news release from RNA therapeutics company ProQR Therapeutics N.V. ("ProQR"), the first patients have been dosed in two Phase 2/3 clinical trials evaluating QR-421a for USH2a-mediated retinitis…
24-year-old Georgia Hughes was concerned when she noticed that her son Myles, age 3, had missing teeth. After a number of doctor visits and MRIs, Myles was diagnosed with 4H…
According to Charcot-Marie-Tooth News, researchers have associated HINT1 gene mutations with neuropathy in Greek patients with Charcot-Marie-Tooth disease (CMT). In the past, HINT1 was linked to CMT in central and southeastern Europe. However, researchers…
Look out, Nebraska - there may soon be an uptick (see what we did there?) of Lyme disease cases within your state. Just about two years ago, Borrelia burgdorferi (B. burgdorferi),…
In 1960, the FDA approved chlorthalidone for hypertension (high blood pressure). However, doctors were unsure whether this therapy could benefit patients with advanced chronic kidney disease (CKD) who had high…
Currently, the standards-of-care for patients with Hurler syndrome, or one of the forms of mucopolysacchardiosis type I (MPS I), include enzyme replacement therapy and hematopoietic stem cell transplants. However, Medical…
It is no secret that medical research can provide crucial insights into disease pathology, prognostic factors, and patient outcomes. Recently, a research team from Hokkaido University in Sapporo, Japan dove…
In early December 2021, specialty pharmaceutical company Aytu BioPharma, Inc. ("Aytu") announced that its therapy AR101 (enzastaurin) earned Orphan Drug designation from the FDA. According to the announcement, the therapy…
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