The task of letting go starts at the time of diagnosis of the rare disease, when families have to let go of the dreams and expectations they had for the…
According to a publication from FOXBusiness, Waltham, Massachusetts-based Thermo Fisher Scientific Inc. has agreed to buy out Brammer Bio for $1.7 billion. With the acquisition, Thermo Fisher, a lab equipment…
Fabry Disease Fabry Disease is a rare lysosomal storage condition. Mutations in the GLA gene cause a type of fat called globotriaosylceramide to build up in the body's cells. Symptoms…
"The simple idea of a single gene leading to a single disease is more likely to be an exception than a rule." Finding the Disease-Causing Genes Things would be, not…
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A Rare Intervention For His Children’s Rare Disease An inspiring article has recently been published in the journal Nature that credits Nick Sireau, a resident of the UK, with…
According to a publication from Medical News Today, in animal studies, a failed cancer drug showed signs of promise in treating frontotemporal dementia. Frontotemporal Dementia Dementia is a blanket term…
Zachary and Imogen Jones are brother and sister with cystic fibrosis (CF). While Imogen is older, she received her CF diagnosis after her brother. It was actually through educational programs…
According to a story from Parkinson's News Today, April is Parkinson's Disease Awareness Month. This year, special focus will be given to patients with the rare, early onset form of…
According to a publication from EurekAlert, results from a phase 1b clinical study of experimental glioblastoma “vaccine“ IGV-001 suggest that the treatment is well tolerated in humans. Additional data collected…
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According to a recent article from EurekAlert!, researchers used data from the Swedish Twin Registry and found that most of the 7 diseases they were studying were inherited. The second finding…
The FDA previously announced their plan to hire 50 new employees specifically to manage the influx of gene therapy applications. Currently, they have approximately 800 applications awaiting review and they…
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Three cases of fibrodysplasia ossificans progressiva (FOP) a rare genetic disease, were reported recently in Stat’s health issue. Wendy’s Story Wendy Weldon was a brave little nine years old when she…
According to the publication from RedlionTrader, a second man infected with HIV seems to have been cleared of the infection, those scientists remain highly cautious of touting the case as…
A New Partnership Patient Worthy is honored and excited to announce our new partnership with The B.L.A.I.R. Connection. The B.L.A.I.R. Connection was founded by Grey Chapin who knows firsthand what…
According to a story from BioPharma Dive, an ongoing discussion in medical circles, both rare and otherwise, is improving the design of clinical trials. Clinical trials are an important tool…
A story recently published on the website of British YouTuber Myles Power covered the incredible, unlikely, and infuriating story of two of America's latest great snake oil salesmen - Ernst…
According to a publication from EurekAlert, new research presented at the annual conference of the American Association for Cancer Research (AACR) suggests that a combination therapy of two regularly-administered immunotherapy…
According to a publication from Blood Journal, an experimental beta thalassemia drug called luspatercept yielded positive results at the end of a phase 2 clinical study. Many patients enjoyed an…
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