Written by Lily Sander By the time I was age four, I started experiencing unexplained symptoms such as severely turned in feet, which caused extreme pain and mobility issues. Terrified,…
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…
The damage caused by cocaine can sometimes be misdiagnosed as a rare disease: granulomatosis with polyangiitis. Cocaine use has been increasing in America and the UK, resulting in serious…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
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…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
On April 25, 2023, myTomorrows, a platform designed to help patients obtain access to experimental medicines, recently launched a new tool called TrialSearch AI, an artificial intelligence-powered resource for physicians…
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…
Written by Libby Wilson- Mommy, Living with Stage IV Breast Cancer Dear Violet - Before I met you, I never loved someone with every morsel of my being. I never…
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…
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…
Written by Katherine Tran Lupus is among the most perplexing autoimmune diseases. It can be debilitating when not regulated, with symptoms ranging from butterfly-shaped rashes across the face to fatigue,…
Written by K.M. I have never given much thought to how I would die. Perhaps because my health and longevity has been laid out for me since infancy. Maybe I…
In the last decade, NAFLD in pregnancy has nearly tripled in concert with the worldwide increase in diabetes. Mayo Clinic reports that NAFLD in general affects one-fourth of the…
Marsha Calloway-Campbell lives a full life as a lawyer, business owner, wife to her husband Armaray, mother to her three adult daughters, and “Naun” to her toddler grandson. But her…
Gene therapy has seemed out of reach for decades. Yet advocates have considered it a way to have damaged or missing genes restore dystrophin and other proteins. Doctors and advocates…
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…
For many rare diseases, gene therapy, not traditional therapeutics, is perhaps the only means to significant relief and cure. However, the path to gene therapy has been strewn with obstacles…
A Business Wire Press Release on April 26, 2023, announced positive interim clinical data on an investigational RNAi therapeutic. Alnylam Pharmaceuticals and its partner Regeneron Pharmaceuticals heralded interim results…
Canada’s unemployment rate is at a record low. Industries have recovered 126% of the jobs lost by the pandemic. On the health front, scientific knowledge and the ability to…
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