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.
Currently, there are no known cures for CDKL5 deficiency disorder, a rare genetic disorder characterized by seizures and severe developmental delays. Treatment, rather, is symptomatic and supportive. However, a news…
In the past, some researchers believed that fibromuscular dysplasia was a rare disease. While some researchers estimate that the incidence is actually much higher than reported, there is still a…
Previously, clinical-stage genetic medicines company Homology Medicines, Inc. ("Homology") submitted an Investigational New Drug (IND) application to the FDA for HMI-103, an investigational gene editing therapy designed to treat phenylketonuria…
According to The Guardian, England’s National Health Service (NHS) has announced that a new therapeutic option will be available for patients with sickle cell disease (SCD). This treatment, called crizanlizumab,…
In June 2021, Redx Pharma dosed its first healthy volunteer in a Phase 1 clinical trial evaluating the safety and phamacological profile of RXC007. Altogether, this investigational therapy is designed…
According to a news release in Yahoo! Finance, biopharmaceutical company Zogenix, Inc. recently submitted a Supplemental New Drug Application (sNDA) for its drug candidate FINTEPLA (fenfluramine). The therapy is designed…
At two years old, Charlotte Wilson, known affectionately as Lottie, has such a bright and happy smile. Her family, including her mother Lisa, feels lucky to still have her…
In a news release from September 30, 2021, biopharmaceutical company Arrowhead Pharmaceuticals ("Arrowhead") shared that it had initiated its Phase 2b AROAPOC3-2002 clinical trial and that the first patient…
Fast Track designation is requested by the drug company; this process and designation are designed to facilitate the development and review of therapies which can treat serious or underserved conditions,…
In the past, imatinib has shown promise for treating patients with pulmonary arterial hypertension (PAH). But specialty pharmaceutical company Tenax Therapeutics, Inc. ("Tenax") believed that it could create a specialty,…
Clinical trials can be helpful tools in determining the safety, efficacy, tolerability, recommended dose, and pharmacokinetics of specific drugs (among other things). In doing so, these trials help tell researchers…
Silica is a naturally occurring substance found in various types of rocks, clay, and sand. Unfortunately, when this silica dust is inhaled, especially over long periods of time, it can…
Did you know—October is known as Spina Bifida Awareness Month, with October 25 being designated as World Spina Bifida and Hydrocephalus Day? That means, all October long, you can help…
Sometimes, companies work together to develop the best therapeutic options for patients. According to Optical Prism, biopharmaceutical company reVision Therapeutics, Inc. and specialty pharmaceutical company Aequus Pharmaceuticals Inc. recently joined…
When Rhonda Stevey met her grandson, Koehyn, she was immediately smitten. The grandmother, who lives in Ohio, felt like her grandson was the happiest infant she had ever met. Unfortunately,…
Paul Williamson has always been a fiercely proud father and an advocate for the rare disease community. His son James was born with Smith-Magenis syndrome (SMS), a rare genetic developmental…
For many patients, symptoms of Huntington’s disease appear within midlife: between the 30s and 40s. This progressive disorder causes brain cell death, leading to a loss of physical and mental…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, identify, or diagnose rare or life-threatening conditions. "Rare" conditions are those affecting under 200,000…
To learn more about various conditions, how to treat them, and how to improve patient outcomes, medical research plays a crucial role. According to a news release from Saint Louis…
Prior to October 8, 2021, there were no FDA-approved treatment options for pediatric patients with congenital athymia, an ultra-rare immune disorder. However, this all changed when the FDA approved Rethymic…
Did you know that just one tick bite could transmit to you a number of infections? That's right, according to a Lyme Science blog post from Dr. Daniel Cameron, MD,…
From October 7-11, 2021, the National Spasmodic Dysphonia Association (NSDA) is holding their 2nd annual Walk for Talk fundraiser to help raise awareness around, and funds for, spasmodic dysphonia, a chronic…
The 2021 Society for Hematological Oncology (SOHO) Annual Meeting took place last month from September 8-11, 2021. During the meeting, researchers discussed new advances in the field of hematology. According…
With each new day and technological improvements come new discoveries about the world around us. Some of those discoveries center around medical conditions which have never been seen before. According…
According to the Daily Sabah, the Health Ministry of Turkey is taking a new approach to family planning and screening for genetic disorders. As of October 8, 2021, the Health…
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