Spinal muscular atrophy (SMA) is a genetic condition which affects between 10,000 and 25,000 in the United States. While rare, it is currently the leading genetic cause of infant mortality. But,…
Nonalcoholic fatty liver disease (NAFLD) is a condition which causes fat to accumulate in the liver. Nonalcoholic steatohepatitis (NASH) is a severe form of the disease in which the patient also…
Canavan disease is a type of leukodystrophy frequently found in the Ashkenazi Jewish population. It's a rare genetic condition which is ultimately fatal. It's all caused by a deficiency of the aspartoacylase…
Emily Sadler is only 15. In March of 2017 she was diagnosed with thyroid cancer. This came as a huge shock to herself and loved ones, especially because thyroid cancer…
The government shutdown hasn't had any startling affects on rare disease research yet, but drug developers have voiced their concern that it could soon begin to hinder the research process.…
Happy Thursday! We hope everyone's week is going well. Today, we're highlighting a contribution from a cystinosis patient on the necessity of third-party medical providers. We're also sharing a story…
Xconomy.com has just released that funds have been raised for a variety of companies investigating therapies for rare conditions. Unfortunately, novel therapies and inventions cost million of dollars to develop. Thankfully,…
Myelodysplastic syndromes (MDS) is the name used to describe a group of stem cell disorders in which the body can't produce an adequate amount of platelets and blood cells. Currently,…
Being diagnosed with any serious condition can be scary, overwhelming, and full of unknowns. For those who are diagnosed with a rare disease such as Duchenne muscular dystrophy, spinal muscular atrophy,…
Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It's just been published that in 2018 they approved a…
While medical students may go into the profession dwelling on the lives they can save, the inevitable fact of the profession is that there will be times when they…
Patients with acquired thrombotic thrombocytopenic purpura (aTTP) showed a significantly improved platelet count response after treatment with Cablivi® according to the New England Journal of Medicine's Phase 3 trial results. A…
According to an article recently published by Forbes, one data scientist wants to explain why data science has proven to be slow at identifying particular biomarkers in medical patients. Imran…
As discussed in a recent article on PatientWorthy the FDNA has recently developed a new kind of algorithm which can diagnose rare genetic conditions such as Angelman syndrome, Williams syndrome, and Fragile X syndrome using…
At age twenty one, Lisa was about to graduate college and had a promising future career in front of her. All that was left was graduation itself. The week before…
"We want to have better ways to figure out who is non-adherent so we can focus our efforts better on those patients who may require more assistance or specific resources…
Perhaps the most difficult thing about rare disease research is that not only do the conditions affect only a small portion of the population, but like every diagnosis, they affect…
According to a story from PR Newswire, pancreatic cancer rates in the US are increasing by almost two percent each year. This puts the disease in position to become the…
Juvenile idiopathic arthritis, Lupus, and Malaria are diseases which currently have limited treatment options. But a new discovery by researchers at the Benaroya Research Institute (BRI) could potentially lead to…
According to a story from New Scientist, a team of scientists at the Institute of Cancer Research in London were able to develop an AI tool that was able to…
According to a story from BioPortfolio, the Bristol-Myers Squibb Company recently announced that the European Commission has recently approved the first combination treatment for renal cell carcinoma available in the…
A January 4th post by the Lancet's EBioMedicine headlined the first-in-human pilot study results published by UT Health San Antonio in collaboration with the Mayo Clinic and the Wake Forest School…
According to a story from news-medical.net, a team of researchers from UCLA have successfully developed an approach that allows for the reversal of the mutation that is responsible for the…
A free MDS Patient & Family/Caregiver Forum, sponsored by The MDS Foundation Inc., will be held in Beverly Hills, California on February 2nd. When: Saturday, February 2nd, 2019 Where: Beverly…
For those with a complicated medical diagnosis, state-funded health insurance bodes little practicality. Patients living with rare and chronic disease must schedule visits to multiple specialty clinics and physicians, especially…
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