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.
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.…
Continue ReadingEarly Data Suggests Lenzilumab Benefits for CMML
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…
Continue ReadingMRI Can Help with Earlier CKD Identification and Diagnosis
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…
Continue ReadingBariatric Surgery Could Improve Outcomes for Obese Individuals with NAFLD
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…
Continue ReadingBoy with Batten Disease Pens Book About Marshmallow, His Cane
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…
Continue ReadingMom Runs in Flying Pig Marathon to Raise SHINE Syndrome Awareness
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…
Continue ReadingOrganoids Could Be Used to Find Effective HSP Treatments
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…
Continue ReadingICYMI: Two Professors Receive $74K Grant to Study Alexander Disease
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…
Continue ReadingFamily is Fundraising to Afford Therapy for Girl with Transverse Myelitis
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…
Continue ReadingTeen with SMA Uses Art to Raise Rare Disease Awareness
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…
Continue ReadingSIS-101-ADO Granted Orphan Drug Designation for Autosomal Dominant Osteopetrosis Type 2
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…
Continue ReadingRECAP: NORD’s Living Rare, Living Stronger Patient & Family Forum
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…
Continue ReadingElfabrio Now Approved for Adults with Fabry Disease
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…
Continue ReadingAggies’ Head Coach Jimbo Fisher Says Gene Therapy Could Cure Son’s Fanconi Anemia
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…
Continue ReadingRucosopasem Manganese Granted Orphan Drug Designation for Pancreatic Cancer
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…
Continue ReadingPrimary Endpoint Met in Sepiapterin Trial for PKU
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…
Continue ReadingHydroxyurea is Recommended for Children with Sickle Cell Anemia. So Why Does It Remain Underused?
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…
Continue ReadingStudy Shows How Setmelanotide Reduces Hyperphagia Associated with Bardet-Biedl Syndrome
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…
Continue ReadingPYX-201 Granted Orphan Drug Designation for the Treatment of Pancreatic Cancer
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…
Continue ReadingOCU410ST Granted Orphan Drug Designation for ABCA4-Associated Retinopathies
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…
Continue ReadingUsing Teleretinal Imaging Could Improve 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…
Continue ReadingTwo Cases of Eastern Equine Encephalitis (EEE) Identified in Polk County, FL
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…
Continue ReadingWhy are KRAS-Mutated Colorectal Cancer Tumors More Difficult to Treat?
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…
Continue ReadingRare Community Profiles: From the 1960s to Now: How Two Hemophilia Advocates Have Seen Treatment and Research Progress
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…
Continue ReadingRare Community Profiles: How Derrick’s Colorectal Cancer Journey Led Him to Advocate for Increased Biomarker Testing
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