People with spinal muscular atrophy and other rare conditions with either few or no approved treatments have recently had their hope of a cure renewed by the concept of gene therapy. Basically,…
According to a story from Science and Technology Research News, a group of researchers from the Cleveland Clinic led by Dr. Aziz Nazha will soon present a new myelodysplastic syndromes…
January is Cervical Health Awareness Month! So, why not use the momentum and goal-making spirit of New Year's Resolutions to make sure you get screened for cervical cancer and schedule…
Check out part one of Seth's story here. Seth had set a google alert for his hometown, Natick, to stay up to date in his home's local news. One day,…
Cold agglutinin disease (CAD) occurs when the body's antibodies destroy their own red blood cells. It is a form of hemolytic anemia, meaning the body can't produce enough blood cells fast…
According to a story from Pharmaphorum, there are a number of new treatments that have been introduced or have seen major strides in development over the last year. More patient…
What if you could prevent all of the world's most fatal genetic diseases? According to Lee D. Cooper, there's a way- we're just not educating people on how to do…
While rare disease affects people all around us, often the mainstream press . Which is why it's extra important when a news outlet as big as the Los Angeles Times…
This wonderful story was brought to Patient Worthy by our partners, the Cystinosis Research Network (CRN). The season of giving started off in an unconventional setting for the Moore Family. Chandler…
When Seth was twelve years old, he and the rest of his family began to realize something was wrong with his mother. Seth can remember conversing with her pleasantly, and…
According to a story from sectorpublishingintelligence.co.uk, the clinical stage pharmaceutical company Retrotope recently announced that the first patient had been dosed in its Phase 2/3 clinical trial. The trial will…
A GoFundMe page has been set up for two-year-old boy Kaiden, who was recently diagnosed with a rare vascular condition known as Moyamoya disease. His family is trying to raise…
On Christmas Eve, Josiah Viera passed away at the age of 14, after a public, inspiring journey with the extremely rare disease progeria, which causes accelerated aging in children. Josiah was…
Purdue University just reported that researchers have identified a biomarker (a measurable substance in an organism whose presence is indicative of disease or infection) that could be an early sign…
Dermatomyositis (DM) is a disease which causes muscle weakness and rashes on the skin. Immunosuppressive drugs are a common treatment prescribed for the condition. Patients may also be given methotrexate, rituximab,…
Japanese drugmaker Chugai now has two positive phase 3 studies backing the efficacy and safety of the antibody satralizumab to treat neuromyelitis optica spectrum disorder (NMOSD). Great news for the patient community of…
The views and opinions expressed in this article are those of the authors and do not necessarily reflect the official policy or position of Patient Worthy. According to a story from Vox, a review of over…
Pantothenate kinase-associated neurodegeneration (PKAN) is a neurodegenerative disorder caused by a mutated PANK2 gene. This mutation disrupts a metabolic pathway, decreasing the body's levels of CoA. CoA, or coenzyme A,…
According to a story from Scoop Independent News, citizens of New Zealand that have the rare bleeding disorder hemophilia A will soon have access to the drug emicizumab (marketed as…
There is some promising news coming out of the American College of Asthma, Allergy and Immunology (ACAAI) annual meeting in Seattle, Washington. According to a case study, rare forms of inducible urticarias…
The display at my local gas station is an impressive one. CBD oils - from no fewer than three different manufacturers and in half a dozen flavors - are packed,…
As it turns out, I am obsessed with Scrubs. The TV show, not the song. When I was at my sickest to date, those TV episodes got me through a…
According to a story from globenewswire.com, the gene therapy company AVROBIO recently announced that the company's experimental gene therapy drug candidate AVR-RD-01 has earned Orphan Drug designation from the US…
Big news out of the FDA last week! Alexion Pharmaceuticals got approval for ALXN1210 for paroxysmal nocturnal hemoglobinuria, or PNH, which will now be sold as Ultomiris, while Stemline got approval…
According to a story from BioSpace, the biopharmaceutical company Merck recently announced that the US Food and Drug Administration (FDA) has approved the company's cancer drug KEYTRUDA in a new…
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