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 the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. A rare disease is one…
Continue ReadingSerdexmethylphenidate for Idiopathic Hypersomnia Earns Orphan Drug Designation
The American Association for the Study of Liver Disease (AASLD) held its annual Liver Meeting from November 4-7, 2022 in Washington, D.C. During the meeting, Mirum Pharmaceuticals shared two late-breaker…
Continue ReadingINTERVIEW: Mirum’s Chris Peetz Discusses Positive Topline Data on LIVMARLI for PFIC (Pt. 1)
Annie and Peter McKenzie describe their seven-year-old son Patterson as having a “heart of gold.” Patterson is bright, funny, happy, and empathetic. Despite his insistence that he is not a…
Continue ReadingRaising Awareness of Recessive Dystrophic Epidermolysis Bullosa: Patterson’s Story (Pt. 1)
Have researchers from Rice University and Baylor College of Medicine discovered a potential treatment option for mesothelioma? According to a news release from the U.S. National Science Foundation, who…
Continue ReadingICYMI: Advanced Mesothelioma Eradicated in Mice Models
Before you read further, make sure to check out Part 1 of our interview, where Brittany discusses what non-small cell lung cancer (NSCLC) is, her diagnostic journey, and how she learned that…
Continue ReadingAdvocating for Cancer Biomarker Testing: How Brittany Turned her RET+ NSCLC Diagnosis into Action (Pt. 2)
Since her son Tobias was born, Sophia Rincon has had to embrace her new role as medical mom. According to an article in KENS5, Tobias was born with congenital diaphragmatic…
Researchers have been evaluating '1104, an investigational immune-resetting molecule, for patients with eosinophilic esophagitis (EoE). Through immune resetting, some believe that '1104 could reduce the inflammatory cascade related to…
On the one-year anniversary of her RET+ non-small cell lung cancer (NSCLC) diagnosis, 37-year-old Brittany Hawkins had spent some time reflecting on her journey. When she was just seventeen years…
Continue ReadingAdvocating for Cancer Biomarker Testing: How Brittany Turned her RET+ NSCLC Diagnosis into Action (Pt. 1)
According to an October 31, 2022 press release from muscle disease company Dyne Therapeutics, Inc. ("Dyne"), the company's therapeutic candidate DYNE-251 earned Fast Track designation from the FDA for Duchenne…
Over the past decade, there has been growing research into the effects of various chemicals, like herbicides or pesticides, on human health. Researchers want to understand how herbicide or pesticide…
An estimated 4 million adults within the U.S. have fibromyalgia, a chronic disorder characterized by fatigue, problems with sleep and memory, and musculoskeletal problems. Prior studies have found that a…
Continue ReadingGreen-Light Glasses Could Reduce Fibromyalgia Related Pain and Anxiety
Over the years, there has been more research into the impact of diet and nutrition on our overall health. How does what we eat – and how it was prepared…
Continue ReadingEating Less Processed Food Could Reduce Crohn’s Disease Risk
Is there any relationship between achalasia and esophageal cancer? According to Healio, a research study found that there does seem to be some level of correlation. In Abstract S388, presented…
Continue ReadingAchalasia Linked to Increased Esophageal Cancer Risk
From October 7-11, 2022, the AAPNational Conference & Exhibition offered insight into clinical practices, trends, and other important information within the pediatrics specialty. According to Contemporary Pediatrics, the Family Heart…
The current standard-of-care for Fabry disease is called enzyme replacement therapy (ERT). According to Fabry Disease News, researchers have been working to evaluate an experimental ERT for patients called ISU303.…
Familial hypercholesterolemia (FH) is an inherited form of very high cholesterol. Early treatment is needed for those living with FH to lower cholesterol levels and reduce the risk of heart…
Continue ReadingCroatia to Evaluate Students for Familial Hypercholesterolemia
Researchers and scientists have stated that it can be difficult to diagnose and treat patients with mucinous ovarian cancer. In fact, because the cancer is often not found until it…
Continue ReadingResearchers Build Deeper Understanding of Mucinous Ovarian Cancer
Growing up, Matthew Gee always seemed to love football. He played as a linebacker for the 1989 Trojans at the University of Southern California and was part of the 1990…
Each day, an estimated five children within the U.S. are born with congenital diaphragmatic hernia (CDH), a diaphragmatic defect which is fatal in approximately 33% of cases. While these hernias…
It’s no secret that more mathematics, data, and technology are being used to drive advances within healthcare and medicine. For example, we’ve seen the heightened use of artificial intelligence (AI)…
Continue ReadingMathematical Model Being Developed to Evaluate Esophageal Cancer Progression
What is the underlying cause of cancers such as esophageal adenocarcinoma or intestinal gastric cancer? Are there certain precursor cells that these cancers arise from? According to a news release…
Continue ReadingResearchers Identify Precursors to Esophageal and Gastric Cancer
More rare conditions are discovered as we learn more about the human body and genetics. For example, shares an article in Medical XPress, a research team from the University of…
Clinical trials play a crucial role in better understanding rare diseases and therapeutic interventions. According to a news release from rare disease company Albireo Pharma, Inc. (“Albireo”), enrollment is now…
Continue ReadingEnrollment Complete in Bylvay Study for Biliary Atresia
Oxytocin has been deemed the "love drug" or "love hormone." This hormone, which controls aspects of behavior and reproduction, is often released when we're in love or in physical…
Continue ReadingIntranasal Oxytocin Derivative Could Reduce Alzheimer’s Cognitive Impairment
According to a late October 2022 news release from gene therapy company AVROBIO, the sixth patient has been dosed in a Phase 1/2 clinical study evaluating AVR-RD-04 for patients…
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