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…
It is believed that in 1916 a German doctor named Brachmann first identified a patient with symptoms of a disease later known as Cornelia de Lange syndrome. Then in…
A recent article emphasized the importance of recognizing symptoms that may mean you have meningitis, including headaches. Meningitis Meningitis is an inflammation of the fluid and membranes (meninges) surrounding your…
Galecto has announced they have completed enrollment in their Phase2b trial which will investigate GB0139 as a treatment for idiopathic pulmonary fibrosis (IPF). This trial is called GALACTIC-1. Galecto anticipates…
A recent article in Labroots stated that the majority of patients who have preleukemic disorders may not be candidates for leukemia. Estimates are that there are about ten to fifteen…
On the first Sunday in May, Cincinnati, OH hosts the Flying Pig Marathon – its 26.2 mile race. This year marked the 24th annual event. But for 17-year-old Diego Ramirez,…
Clinical trials are an important tool within the healthcare realm. These trials assist in developing deeper understandings of diseases, assessing and discovering treatment options, and learning new ways of identification…
Aeterna Zentaris’s ongoing DETECT trial is evaluating the drug macimorelin (tradename Macrilen™) for childhood-onset growth hormone deficiency (CGHD). As recently announced in an article published by GlobeNewsWire, the study (AEZS-130-PO2),…
Recently, an international group of pediatric rheumatologists and nephrologists met and created a consensus on a standard for a steroid-dosing regimen to treat childhood-onset systemic lupus erythematosus with proliferative lupus…
This story was originally published in The Cystinosis Advocate, the newsletter for the Cystinosis Research Network, a Patient Worthy partner organization. Continued From Part Two We were told about what…
The Food and Drug Administration (FDA) has four distinct approaches to expedite the availability of therapies for rare, severe, or life-threatening illnesses: Priority Review, Breakthrough Therapy, Fast Track, and Accelerated…
The month of May is recognized as National Myositis Awareness Month, a time to spread awareness about myositis among the general public and the medical field. Organized by The Myositis…
After over 30 years of not knowing another family dealing with the same rare condition that our daughter Kelley had, we finally started to connect with others, thanks to the availability of…
For the last 52 years, individuals with Wilson disease have been relying, largely, on the same treatment options. These options, designed to remove copper from the body, include Cuprimine (penicillamine).…
In Europe, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare, life-threatening, or chronically debilitating conditions. For the European Union (EU), a “rare”…
Imagine the highs and lows that accompany the doctor’s announcement that your sweet, innocent two-year-old daughter has leukemia. The good news is that your daughter has the type of leukemia…
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…
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