Vogmask is a San Francisco, California based company founded in 2011. They create stylish facial masks to protect wearers from inhalation of particulate and allergens. These types of masks can…
Bontulinum Neurotoxins (BoNTs) are a protein that are used to treat a wide array of conditions including cervical dystonia, blepharospasm, neurogenic detrusor overactivity, and sialorrhea to name a few. These proteins…
The development of gene therapy as a treatment for rare and life-threatening conditions is a quickly growing area of scientific exploration. As more drug developers have begun investing in this…
Cystinosis Cystinosis is a rare disease which is caused by a buildup of cystine in the body's cells. This buildup impacts various organs including the brain, eyes, liver, kidneys, and muscles.…
Mucolipidosis Type IV Mucolipidosis Type IV (ML-IV) is a rare disease which causes a reduced mental capacity and blindness. ML-IV is caused by a missing or malfunctioning TRPMLI protein. Most patients never…
Cameron Dixon is a 17-year-old boy living with muscular dystrophy. His family was first told that he would most likely never be able to walk, eat, or talk, and that…
Precision mNedicine is the latest craze in rare disease research. Thankfully for patients, it is completely, 100% centered on improving the quality of their care. Unlike most typical healthcare practices,…
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…
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.…
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…
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…
"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…
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…
The Right-To-Try Law is a federal legislation which allows terminally ill patients the opportunity to use experimental drugs that don't yet have FDA approval. It's controversial at best, but some…
Merkel cell carcinoma (MCC) is a form of skin cancer. In the United States, about 2,500 people are diagnosed with MCC each year. The condition is treated with chemotherapy, immune…
FDNA has created a new type of algorithm which may be able to help diagnose rare genetic conditions like Noonan Syndrome by a simple picture of a person’s face. What’s particularly…
Every day the scientific community makes progress in the study of rare disease. Research in the field has advanced exponentially in recent years and we're starting to see more treatments…
We tend to daydream about miracle cures for rare and detrimental conditions like ovarian cancer. Sometimes they exist, but often it's more of a battle to find the right combination…
A recent study conducted in Oregon has shown that emergency medical responders may be biased against minority patients. It's the concept of unconscious bias. Discrimination is illegal, but that doesn't…
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