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 March 2023, the European Commission (EC) approved Pombiliti (cipaglucosidase alfa) for the treatment of adult patients living with late-onset Pompe disease. Just three months later in June, another…
In 2019, the Baird family, living in Scotland, received news that nobody wants to hear. Michael, the family patriarch, was diagnosed with a gastrointestinal stromal tumor (GIST), an uncommon tumor…
Following allogeneic hematopoietic stem cell transplant (allo-HCT), which uses healthy stem cells from a donor, many doctors prescribe antibiotics to patients as a protective measure. Many individuals become neutropenic…
How does hypoparathyroidism evolve and progress over time? Are available treatment and management strategies providing efficacious disease control? What are the long-term effects of hypoparathyroidism on overall health—and are there…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Sometimes it can be difficult to target and treat cancer without harming healthy tissue. Even while surgically removing tumors, it is possible to accidentally hurt or damage healthy tissue,…
14 touchdowns. 11.6 yards per carry (on average). 1st Team All-Conference. These accolades show how hard Luke Winterbottom, an all-league running back for Plymouth Whitemarsh High School, worked during…
You might know Danny Bonaduce for his role as Danny Partridge in "The Partridge Family” or his time as an American radio personality. But Bonaduce can now also call himself…
Opdivo (nivolumab) is an immune checkpoint inhibitor that binds to PD-1 and contributes to cancer cell death. It has shown efficacy in treating a number of cancer types, both…
The World Health Organization (WHO) reports that an estimated 3.23 million people worldwide die each year from chronic obstructive pulmonary disease (COPD), making it the third leading cause of…
Every parent hopes that their child will be born safely and healthily. But for one family in India, their daughter’s birth brought a host of concerns about her health. From…
From the time he was born until now, 6-year-old Jawn Fisher has been entranced by law enforcement. He loved the uniforms, the fight for justice, and the badges that…
In the past, doctors have noticed a trend in hepatocellular carcinoma (HCC). More specifically, research has shown that HCC can grow resistant to certain drugs. This makes it more…
The American Society for Clinical Oncology (ASCO) held its Annual Meeting in late May and early June to discuss clinical guidelines, best practices, and research advancements within the oncology…
The Orphan Drug Act of 1983 was implemented after the U.S. Food and Drug Administration observed drug development issues in the rare disease space. Rare diseases often have smaller…
Both Ofev (nintedanib) and Esbriet (pirfenidone) are FDA-approved to treat individuals living with mild, moderate, or severe idiopathic pulmonary fibrosis (IPF). The treatments work by preventing fibrosis (scarring) and stopping…
When it comes to advancing treatments and research, collaboration is key. A new partnership recently formed; this collaborative effort underscores the need for additional support and resources for people living…
As she stood in the garden, surrounded by friends and family, a splitting headache richocheted through Ciara Wilkie's head. Her vision blurred. At first, Ciara didn't think much of it.…
When it comes to treating interstitial cystitis, doctors may take a multipronged approach: physical therapy, bladder distention, surgery, nerve stimulation, oral medications. But people with this condition lack therapies…
The U.S. Food and Drug Administration offers various designations to drugs and drug developers to both incentivize medical advancements in the rare disease space and get treatments in the…
In 2015, rare disease stakeholders in North Carolina established the first Rare Disease Advisory Council (RDAC); the National Organization for Rare Disorders (NORD) explains that an RDAC is an…
As recently reported by Forbes, the U.S. Food and Drug Administration (FDA) approved Leqembi (lecanemab) for Alzheimer’s disease earlier this week. Leqembi is a humanized monoclonal antibody that targets…
In the average person, you would expect to see normal to near-optimal low-density lipoprotein (LDL) cholesterol levels sitting at 129 mg/DL or lower; levels under 100 are seen as healthier.…
For the first year and a half of her life, Audrey seemed to be a healthy child. Her parents Tenneil and Tom had no real concerns about her health. So…
When biopharmaceutical company Invex Therapeutics (“Invex”) first decided to launch a clinical trial to evaluate Presendin (sustained-release exenatide) for idiopathic intracranial hypertension (IIH), the company felt excited. Presendin is a…
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