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 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…
Dimeric calpains are calcium-activated cysteine proteases which help to regulate cell function. If these calpains are overactive, it could lead to fibrosis (tissue scarring) and tissue damage. In the…
Most patients with Norrie disease, a rare X-linked genetic disorder, often develop sensorineural hearing loss. But what mechanisms cause this hearing loss to occur? According to Medical XPress, researchers from…
At just 18 weeks old, Ivy Gaines, who was born prematurely, was diagnosed with spina bifida. His parents, Robert and Brittney, have worked tirelessly to ensure that Ivy is receiving…
The Child Growth Foundation (CGF) is a leading UK charity which supports families, adults, and children with rare growth conditions such as SHOX deficiency, Silver Russell Syndrome, and Sotos syndrome.…
Fenchol, a natural compound, is known for its herbal and appealing scent. This compound is found in a number of plants, including basil, nutmeg, and eucalyptus leaves. In the past,…
To best address the needs of patients with rare diseases and rare cancers, additional treatment options are necessary. But developing these treatment options can be time-consuming and sometimes difficult.…
Screening tests are used to identify potential signs related to certain diseases or conditions. For example, newborn screening, performed at birth, gives insight into whether or not a child has…
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