Personalized Medicine You may have heard of personalized medicine. It's a new wave of healthcare that aims to provide individualized treatment to patients based on their own genetic makeup. The…
Researchers from Brigham and Women's Hospital recently announced a new discovery which could potentially lead to a new multiple sclerosis (MS) therapy. This research has been published in Cell Host & microRNA.…
Therapix Biosciences specializes in cannabinoid pharmaceuticals. Currently, they are working on developing a therapy called tetrahydrocannabinol or THX-110 as a treatment for Tourette syndrome (TS). Additionally, it is being developed…
Some scientists never have the opportunity to meet a single patient in the community of the disease they work their entire lives trying to understand. This can make them feel…
A new precision medicine center, specifically for pediatrics, has launched in Utah. This new center is the result of collaborative efforts from the Intermountain Primary Children's Hospital, Intermountain Precision Genomics,…
The University of Massachusetts Medical School has recently announced preliminary results from their trial for Tay-Sachs disease. This trial is investigating a gene therapy and researchers are excited about it's…
Pulmonary Hypertension Association The Pulmonary Hypertension Association (PHA) is headquartered in Silver Spring, Maryland. It was formed in 1991 by 4 women passionate about finding a cure. Additionally, they hope…
The medical field is constantly evolving, and rightly so. We should never be complacent with things as they are when it comes to healthcare. There is always more to be…
Spinal muscular atrophy (SMA) is a rare disorder, but sadly, it is still the most common genetic reason that children die before the age of 2. Not only that, but…
A team of cross-disciplinary researchers at Arizona State University (ASU) is working to develop a software which patients with neurological disorders may easily use to assess their disease progression. Through…
Phase 3 Trial Results from the Phase 3 PREVENT study for neuromyelitis optica spectrum disorder (NMOSD) were extremely positive. 98% of patients in the treatment group did not experience a…
An amendment is being planned for an ongoing Phase 1/2 clinical trial for amyotrophic lateral sclerosis (ALS). This intention has just been announced by the cell therapy company Kadimastem. However,…
It wasn’t until the 1980s that the medical field really started talking about the need to emotionally support patients, especially those with chronic illnesses. This is partly due to the…
The Bettuchi's Amanda Bettuchi and her husband have 3 children. Their middle child, Shane, was born with severe combined immunodeficiency (SCID). Essentially, this means he was born with practically no…
Researchers at the University of Pennsylvania are working on a new potential gene therapy for Duchenne muscular dystrophy (DMD). DMD is a rare form of muscular dystrophy that primarily affects…
WHIM Syndrome WHIM syndrome is a form of primary immunodeficiency. It is caused by a mutation in the CXCR4 gene. WHIM stands for Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. Throughout their…
Cancer tumors need an abundance of different nutrients to grow. Therefore it would make sense that by cutting off the food supply to cancer tumors, we could successfully inhibit their…
A New Study A recent study published in Nature Immunology and conducted by researchers at the University of Basel has produced influential results regarding primary immunodeficiency disorders (PID). These rare disorders…
The Bio Patient and Health Advocacy Summit of 2019 was recently held in Washington D.C. One of the panels addressed patient engagement with the FDA and how patients, advocates, families, and organizations…
Geisinger was founded by Abigail Geisinger over 100 years ago. It now has 13 hospitals and 2 research centers. They just released a new study examining the severity of symptoms…
Elena Frid is neurologist and Lyme disease specialist. She lives in New York City. After opening her neurology practice in Manhattan, she quickly became aware of some huge issues many…
Firdapse, or amifampridine phosphate, is a medication currently approved for Lambert-Eaton myasthenic syndrome, or LEMS. However, Catalyst Pharmaceuticals has been working to evaluate if it can aid other neurological and…
"We went from being able to treat no patients to being able to treat nearly all patients in seven years." 90% of cystic fibrosis (CF) patients now have a new treatment…
FA Fanconi anemia (FA) is a rare disease in which the bone marrow in the body essentially fails. It is a genetic condition where the body progressively becomes deficient in…
GlaxoSmithKline (GSK) has just announced that Zejula, otherwise known as niraparib, has just been approved for cancer patients beyond those who have a BRCA+ mutation. The patients now included in…
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