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.
Rare Pediatric Disease designation is a status granted by the FDA to drugs or biologics intended to treat rare diseases (affecting under 200,000 Americans) in patients aged 18 or younger.…
Typically, idiopathic intracranial hypertension (IIH) is associated with obesity, or a body mass index (BMI) over 30.0. As obesity rates have continued to increase globally, so have the incidences of…
Previously, Ocaliva (obeticholic acid) was approved as a prescription treatment for patients with primary biliary cholangitis (PBC), a rare and chronic liver disease. However, Market Screener reports that biopharmaceutical company…
At just four years old, Gabriel Spiro is facing his 3rd open-heart surgery. You see, Gabriel was born with hypoplastic left heart syndrome (HLHS), a rare and severe congenital heart…
Prior to 2021, there were no FDA-approved treatments for patients with type 1 plasminogen deficiency, or hypoplasminogenemia. As a result, there was an urgent need to fill this unmet need…
In late May 2021, biopharmaceutical company PTC Therapeutics, Inc. ("PTC") shared that its therapy, PTC923, received Orphan Drug designation within both the United States and Europe. The treatment is designed…
In a press release from late May 2021, biotechnology company CancerVAX, Inc. ("CancerVAX"), which works to develop targeted immunotherapy treatments, shared that it had entered into a sponsored research agreement…
From June 5-8, 2021, the European Renal Association - European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Congress. Though the event was held virtually, the meeting still expanded…
According to a June 11 press release from biotechnology company Equillium, Inc. ("Equillium"), positive topline data is now available from the Phase 1b EQUATE clinical trial. Within the clinical trial,…
Currently, the European Hematology Association (EHA) Virtual Congress 2021 is going on from June 9 through 17, 2021. During the Congress, hematologists and other stakeholders will discuss clinical research and…
Currently, the European Hematology Association (EHA) is holding its 2021 Virtual Congress through June 17, 2021. During the event, researchers are expounding on new insights and research within the hematological…
On June 8, 2021, pharmaceutical and biotechnology company Pfizer Inc. ("Pfizer") shared that its Pneumococcal 20-valent Conjugate Vaccine (PREVNAR 20) is now FDA-approved. The vaccine helps protect against 20 different…
In a news release from June 2, 2021, biopharmaceutical company Soleno Therapeutics, Inc. ("Soleno") shared an exciting update. The company's investigational treatment, DCCR (diazoxide choline) Extended-Release tablets, received Orphan Drug…
At one point in the past, researchers studied a patient with congenital heart disease (CHD) and came out with a potential cause: SORBS2 gene mutations. However, because the study sample was so…
In a news release from June 9, 2021, biotechnology company Vertex Pharmaceuticals, Inc. ("Vertex") shared that its therapy TRIKAFTA (elexacaftor/texacaftor/ivacaftor and ivacaftor) was approved for expanded use. Now, the treatment…
As of June 7, 2021, an injectable treatment is now available for patients aged 1 month+ with paroxysmal nocturnal hemoglobinuria. The FDA recently approved Ultomiris (ravulizumab-cwvz) for these patients. Previously,…
In early June 2021, SPG302, an investigational therapy for patients with amyotrophic lateral sclerosis (ALS), received Orphan Drug designation from the FDA. According to ALS News Today, the treatment…
From June 2-5, 2021, the European Alliance of Associations for Rheumatology (EULAR) held its EULAR 2021 Virtual Congress. During the event, participants discussed basic, translational, and clinical science within the…
According to a recent news release from biopharmaceutical company Arena Pharmaceuticals, Inc. ("Arena"), the company's investigational therapy, etrasimod, received Orphan Drug designation from the FDA. Currently, Arena is evaluating etrasimod…
For decades, it has been clear that diet plays a role in overall health. But can diet also play a role in whether or not you develop certain conditions -…
Charcot-Marie-Tooth disease (CMT) is associated with a number of genetic mutations. In fact, the different mutations signify specific CMT subtypes, such as CMT1 or CMT3. According to Charcot-Marie-Tooth News,…
For all the coffee lovers who drink as much coffee as I do each day, this one's for you. Recently, a press release from the Icahn School of Medicine at…
Have you ever heard of base editing? This relatively new technology within the field of genome editing offers the potential opportunity to treat a variety of genetic conditions. According to…
In 2020, COVID-19, a novel coronavirus, began impacting the globe. Caused by the SARS-CoV-2 virus, COVID-19 has resulted in 172 million diagnoses and 3.7 million deaths worldwide. While acute kidney…
Early on in 2021, the FDA approved voclosporin as an add-on treatment for patients with lupus nephritis (LN), a frequent complication of lupus. Ultimately, this approval was a positive decision,…
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