We've made a lot of progress in cancer research in recent years. Many novel therapies have been developed for illnesses previously considered untreatable. However, as new treatments have been discovered,…
According to a press release from the biopharmaceutical company Cerecor, Inc., the US Food and Drug Administration (FDA) recently agreed to accept the company's Investigational New Drug (IND) application for its…
According to a story from PR Newswire, the international drug developer Eli Lilly and Company recently announced the results of a Phase 3 clinical trial testing the company's drug LARTRUVO…
Hope everyone is having a wonderful Wednesday! The weather where we are is freezing this week-- but that's a perfect excuse to sit down with a few of our top…
Danon disease is a rare condition caused by a mutated LAMP-2 gene. This gene has three different forms- LAMP-2A, 2B, and 2C. Depending in which variant is mutated, individuals with…
According to a story from BioPortfolio, the biotechnology company Vertex Pharmaceuticals recently announced that the European Commission has approved a label extension for the company's cystic fibrosis drug Orkambi. Under…
aTTP aTTP, or acquired thrombotic thrombocytopenia purpura, is an immune condition which results in severe Thrombocytopenia. Thrombocytopenia means the body doesn't have enough platelets in the blood. In aTTP, it's caused…
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.…
According to a story from Biotecnika, Penn Medicine & Inovio Pharmaceuticals Inc. have recently announced that the US Food and Drug Administration (FDA) has signaled its approval for the first…
According to a story from Fabry Disease News, the drug developer Avrobio recently announced that the US Food and Drug Administration (FDA) has given the company's experimental drug AVR-RD-01 Orphan…
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.…
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,…
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…
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