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.
According to Fragile X News Today, a new registry recently launched within the United States to spur research into gene premutations linked to fragile X syndrome (FXS). Currently, the…
On November 27, biotechnology company Vertex Pharmaceuticals Inc. ("Vertex") announced the approval of SYMKEVI with KALYDECO for the treatment of pediatric patients with cystic fibrosis (CF), says GuruFocus. The…
In many areas of society, from healthcare to big business, companies are turning to artificial intelligence (AI) to solve pressing problems. Within the medical sphere, researchers recently developed an automated,…
In early December, biotechnology company Aeglea BioTherapeutics, Inc. ("Aeglea") announced that its therapy ACN00177 received Rare Pediatric Disease designation from the FDA for the treatment of homocystinuria. Should this drug…
In a recent press release, biopharmaceutical company Akari Therapeutics ("Akari") announced the publication of data on intravitreal nomacopan for patients with autoimmune uveitis. The data was sourced from a 2-year…
Recently, the FDA expanded treatment indications for Gavreto (pralsetinib). According to MedPage Today, the drug was previously indicated for the treatment of patients with lung cancers with RET gene fusions. Now, this…
In a press release from late November, pharmaceutical company BioCryst Pharmaceuticals, Inc. ("BioCryst") announced the publication of data from its APeX-J clinical trial in Allergy. During the trial, which was…
Overall, Galapagos works to develop small-molecule treatments for patients with a variety of conditions. In a recent press release, the company reported positive top-line data from the Phase 2 PINTA…
Obesity is considered to be excess fat accumulation that may lead to health issues. Worldwide, the prevalence of obesity has greatly increased over the years; the World Health Organization (WHO)…
According to GuruFocus, the European Medicines Agency (EMA) recently validated a Marketing Authorization Application (MAA) submitted by biopharmaceutical company Mirum Pharmaceuticals, Inc. ("Mirum"). The MAA was centered around maralixibat, an…
On November 30, 2020, biopharmaceutical company Travere Therapeutics ("Travere") announced that patient enrollment was complete for the Phase 3 DUPLEX clinical trial. Within the trial, researchers will evaluate the safety,…
According to Newswise, the FDA recently approved a new prostate cancer imaging technique developed by researchers at UCLA and UCSF. The imaging technique, called prostate-specific membrane antigen PET imaging (PSMA…
Recently, biopharmaceutical company Acceleron Pharma Inc. ("Acceleron") announced that its investigational therapy ACE-1334 was granted Orphan Drug designation by the FDA. The treatment is designed for patients with systemic…
For the entirety of 2020, our lives have been dominated by COVID-19, the novel coronavirus linked to 64.5 million cases and 1.49 million deaths across the globe. In the United…
In 2019, Japan approved the use of EVRENZO (roxadustat) for patients with anemia of chronic kidney disease (CKD) who were also on dialysis. However, the orally administered therapy was not…
This past November, nearly 9,000 attendees showed up for the American Academy of Ophthalmology (AAO) 2020 Virtual Annual Meeting. During the meeting, biopharmaceutical company Kodiak Sciences Inc. ("Kodiak") announced that…
When many people hear CAR T-cell therapy, they think about a treatment designed to address cancer. Recently, a new study done on mice models has suggested that an experimental form…
According to HCP Live, University of Lübeck researchers recently connected rheumatoid arthritis (RA) and pemphigus, an autoimmune disorder characterized by blisters on the skin and mucous membranes. In fact, researchers…
Recently, scientists and researchers from the UCL Great Ormond Street Institute for Child Health (GOS ICH) and the UCL Cancer Institute worked to create an innovative solution for patients with cancerous…
On November 20, 2020, biopharmaceutical company RedHill Biopharma Ltd. announced the initiation of a Phase 3 clinical trial to study the orally administered RHB-204. The drug is designed to treat…
Recently, biotherapeutics company PureTech Health announced that their Phase 1 trial to evaluate LYT-100 (deupirfenidone) has completed. During the multiple ascending dose and food effect study, researchers analyzed the safety,…
Each year, thousands lose their vision to dominant optic atrophy (DOA), an inherited disease that negatively impacts the optic nerves. However, researchers and scientists from Trinity College Dublin may have…
When you think about genetic testing, what first comes to mind? Is it pancreatic cancer? While this might not be the first thing you consider, a new study suggests that…
This past month, the Ice Bucket Challenge was brought back into the spotlight when co-founder of the ALS Ice Bucket Challenge, Pat Quinn, unfortunately passed away. As the ALS Association…
In a recent press release, biopharmaceutical company Akebia Therapeutics ("Akebia") announced the publication of the Phase 3 INNO2VATE study design and methodology. The publication examines the INNO2VATE trial, which evaluated…
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