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.
At first, the round black dots that Carmen saw spattered across the bathroom sink were frustrating, but not concerning. Since her grandchildren were staying with her, Carmen assumed that something…
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…
The AACR Annual Meeting 2023 took place from April 14-19. During the meeting, stakeholders from across the cancer community—from patients to clinicians—come together to discuss trends, research, and clinical practice.…
What do clinical evaluations, blood tests, urine tests, kidney tissue tests, and imaging tests all have in common? These are all diagnostic measures that doctors may use to diagnose chronic…
Bariatric surgery refers to surgical procedures that can help people with obesity to lose weight when other means have not worked. This type of surgery can help people to live…
7-year-old Peter Heath loves to tell jokes, play with Legos, hang out with his friends, and spend time outside. Guided by his white cane, which Peter aptly named Marshmallow, Peter…
Last year, 35-year-old Jenna Neduchal ran her first Flying Pig Marathon. This annual event serves to unite the community, create an accessible event for all, and raise funds for important…
There are no cures or treatments available for people with hereditary spastic paraplegia (HSP) type 56, or SPG56. While the current standards-of-care focus on symptom relief and mobility assistance, patients…
First described in medical literature in 1949, Alexander disease is an extremely rare neurological disorder and form of leukodystrophy. Despite nearly 74 years having passed since its discovery, there is…
On May 7, 2023, Kimberly Kotar completed a half-marathon. That’s impressive in its own right—only more so when you learn that, due to her transverse myelitis, Kotar was once unable…
In March 2023, 14-year-old Rylie Erbacher visited Washington, D.C. for a one-of-a-kind experience. The teen, who has spinal muscular atrophy (SMA) type 2, was one of just 10 people nationally…
Have you ever heard of the Orphan Drug designation? This designation, created through the Orphan Drug Act and granted by the U.S. Food and Drug Administration (FDA), is used to…
Rinvoq (upadacitinib) is a prescription JAK inhibitor that works by blocking pro-inflammatory signaling within cells. In the past, Rinvoq was approved for indications like rheumatoid arthritis, active psoriatic arthritis and…
The National Organization for Rare Disorders (NORD), a Patient Worthy partner, formed in 1983 to provide support and education to people and families living with rare diseases, advocate for change…
On May 10, 2023, reports EMPR, the U.S. FDA approved Elfabrio (pegunigalsidase alfa-iwxj) for adults who are living with Fabry disease. Developed by Protalix Biotherapeutics in conjunction with Chiesi Global…
In 2014, head coach Jimbo Fisher led the Florida State Seminoles to win the BCS National Championship Game. He’s now head coach of the Texas A&M Aggies, recently deemed by…
On May 18, 2023, Market Watch reported that rucosopasem manganese received Orphan Drug designation. Developed by clinical stage biopharmaceutical company Galera Therapeutics (“Galera”), rucosopasem manganese is largely being developed…
Within the Phase 3 APHENITY study, researchers worked to determine the efficacy of sepiapterin in both adults and children with phenylketonuria (PKU). Formerly known as PTC923, sepiapterin was developed…
In 2014, the National Heart, Lung, and Blood Institute released updated clinical guidelines in reference to the care of children with sickle cell anemia. The updated guidelines recommended the use…
Hyperphagia, or excessive and unrelenting hunger, is a key symptom associated with Bardet-Bield syndrome. This excessive hunger often begins around or before 5 years old and can lead to…
Pyxis Oncology is driven to improve the lives of people living with cancer through the development of innovative immunotherapy and antibody drug conjugate (ADJ) therapies. Their treatment PYX-201, a novel…
ABCA4-associated retinopathies are a group of conditions that cause progressive macular degeneration and vision loss due to ABCA4 mutations. Stargardt disease, cone-rod dystrophy type 3 (CORD3), and retinitis pigmentosa 19…
Our country has an issue when it comes to screening for diabetic retinopathy. In addition to clinics lacking the necessary imaging programs, the compliance rate for diabetic retinopathy screening…
Eastern equine encephalitis (EEE) is a rare condition that can be spread through the bites of infected mosquitos. In a majority of cases, birds are infected with EEE. However, if…
Have you ever thought about your genes and what they do for your body? For example, the KRAS gene—do you know what it does? Normally, KRAS encodes for the production…
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