According to a recent article, the sudden death of Cavan woman, Leah Farrelly, shocked her family, friends, and community after living with Long QT syndrome for 9 years. Long QT…
This story was originally published in The Cystinosis Advocate, the newsletter for the Cystinosis Research Network, a Patient Worthy partner organization. Continued From Part One At this stage, the ED…
Mastocytosis refers to an entire group of disorders. All of these conditions are characterized by a rapid production of mast cells (MCs), which are abnormal. This overproduction can occur in…
From April 22 to April 23, 2022, the CureGRIN Foundation and GRIN Therapeutics conducted their 2022 GRI Virtual Conference, a special online event designed to converge patient families, industry stakeholders,…
Currently, there is no way to determine when a relapse will occur in relapsing and remitting multiple sclerosis (MS), one of the two main forms of MS. However, this may…
Inozyme Pharma has recently announced that the first patient was dosed in its Phase 1/2 clinical trial of INZ-701 in adult patients with ABCC6 Deficiency and the second cohort in…
Raising awareness is extremely important when it comes to rare diseases. After all, rarity typically results in minimal public and medical awareness. That's why patient communities and other dedicated individuals…
Artist and author Emily Suñez has been living with multiple rare conditions for over a decade, including Ehlers-Danlos syndrome and complex regional pain syndrome (CRPS), in addition to more common…
This story was originally published in The Cystinosis Advocate, the newsletter for the Cystinosis Research Network, a Patient Worthy partner organization. Oscar was born in January 2020. He was a…
A new treatment for Rett syndrome is officially being studied in humans in Canada, meaning Canadian patients may have a new, viable treatment option in the future. Called TSHA-102, this…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Rhabdomyosarcoma is a rare form of cancer that affects children. It was recently studied at St. Jude Children's Research Hospital, where a team of medical professionals examined cells that persist…
Dr. Omar P. Haqqani, MD FACS is currently the Chief of Vascular and Endovascular Surgery at the Vascular Health Clinics. He has a keen interest in understanding and addressing vascular…
A recent article shared the story of a woman diagnosed with cystinuria whose life was saved after her younger brother donated one of his kidneys. Cystinuria Cystinuria is a rare…
It’s finally May – and you know what that means! That’s right, this month is Huntington’s disease (HD) Awareness Month. The theme for this month’s HD Awareness Month is #LetsTalkAboutHD.…
Ultragenyx Pharmaceutical Inc. has just announced the first dosing in their Phase 2/3 trial investigating setrusumab (UX143) as a treatment for osteogenesis imperfecta (OI) patients. This trial is called Orbit,…
Lennox-Gastaut syndrome (LGS) is a rare, severe form of epilepsy that is often resistant to anti-seizure medications. This means that patients still experience epileptic episodes despite treatment. Because of this…
Patient Worthy is delighted to announce that the National Society of Genetic Counselors (NSGC) has joined us as an advocacy partner! Genetic counselors play a critical role for individuals, families,…
Rare Pediatric Disease designation is granted by the FDA to drugs or biologics intended to treat life-threatening diseases affecting individuals aged 18 or younger. In this case, rare conditions are…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
As featured recently in the Cancer Therapy Advisor, a study has been conducted on patients diagnosed with metastatic renal cell carcinoma (mRCC), who also have elevated inflammation, and sarcopenia. The…
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…
Al Lupiano, an environmental scientist, recently spoke with Meg Baker of CBS2. Al told her that twenty years ago, he was diagnosed with a brain tumor. The memory and…
When Krista Webb was just two years old, doctors determined that she had some level of hearing loss. Her family was surprised about this; nobody else in her family, and…
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…
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