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.
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…
Allegra Sturdevant, age 21, averages thousands of views on her TikTok videos, many of which describe her experiences with Marfan syndrome. According to an article in The Daily Universe, the…
Recently, Kezar Life Sciences Inc. ("Kezar") released interim data and preliminary results from part two of the Phase 2 MISSION clinical trial. According to BioPharma Journal, the results highlight the…
In the National Football League's My Cause My Cleats campaign, NFL players are able to show their passions beyond the game and represent a cause that is important to them through customized…
It is extremely important to get the right medications into the hands of patients who need them. Recently, the FDA helped do this for pediatric patients (aged 2+) with primary…
Meniere's disease is a rare inner ear disorder characterized by "attacks" of tinnitus, ear pain, ear pressure, and dizziness. While doctors have long pondered the exact cause of this condition,…
In a recent news release, biopharmaceutical companies AbbVie, Inc. and SpringWorks Therapeutics, Inc. ("SpringWorks") shared that the two had entered into a joint clinical trial collaboration agreement. Through this collaboration,…
On December 10, 2021, biotechnology company Active Biotech shared that the first subject was dosed within a Phase 1 clinical trial evaluating laquinimod for non-infectious non-anterior uveitis. What is particularly…
In a recent news release, biopharmaceutical company Aligos Therapeutics, Inc. ("Aligos") shared that the first healthy volunteers were dosed in a Phase 1 clinical trial evaluating ALG-055009 for nonalcoholic steatohepatitis.…
On December 6, 2021, biotechnology company Longeveron Inc. ("Longeveron") announced that its investigational therapeutic candidate Lomecel-B earned Orphan Drug designation from the FDA. The therapy is designed to treat patients…
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