From November 14th-16th, 2019, iPain Summit 2019 took place in Los Angeles, CA at the UCLA's Meyer and Renee Luskin Conference Center. Patient Worthy had the privilege to attend and…
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…
People who live with rare diseases have found that it is often difficult to get a diagnosis. Doctors look to common issues before they consider diseases that affect an…
A study conducted by researchers at UC San Diego has revealed new and helpful information about non-alcoholic steatohepatitis (NASH). This study, led by Dr. Joseph Witztum, focused on a…
According to a story from USA Today, Thanksgiving 2009 was a harrowing time for the Anderson family. About a month earlier, Darrin and Sarah Anderson's oldest son Henry had died…
Global Blood Therapeutics was recently granted priority review by the FDA for their new drug Oxbryta. This drug is meant to treat sickle cell disease, and it is the…
According to a story from biopharmadive.com, the field of gene therapy has gained increasing relevance throughout 2019, and 2020 will likely be a continuation of this trend. An area of…
Finding the correct doctor for treatment can be difficult, especially if one has a rare disease. Because few doctors specialize in rare diseases, they are often spread across the…
According to a blog post from Visante, concerns from patient groups and advocates about the safety of a recently introduced tube feeding connector device called ENfit have caused major consternation…
Eva Batista is just three months old, and her parents describe her as an "expressive, smiley, and happy baby." Eva is one of the youngest people to ever be diagnosed…
According to a story from tradeshownews.vporoom.com, the Muscular Dystrophy Association (MDA) has recently announced the official approval of its Patient-Focused Drug Development (PFDD) meeting by the US Food and Drug…
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…
According to a story from PR Newswire, the US Food and Drug Administration (FDA) has recently granted supplemental approval to the drug acalabrutinib (marketed as Calquence) as a treatment for…
When Lucinka was born, it seemed like everything was all right. But it wasn’t. Lucinka wasn’t doing well, and a whirlwind of examinations began. First, a diagnosis couldn’t be made.…
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Amber’s determination to find a miracle for her son Maxwell is detailed in a recent BuzzFeed article. Maxwell and his twin sister, Riley, were born in March 2017. For…
Narcolepsy is characterized by drowsiness, often during the daytime. This is a symptom that greatly affects the lives of those with narcolepsy, and for a long time no FDA…
According to a story from gurufocus.com, the biopharmaceutical company Scholar Rock has recently announced the release of preliminary results from a phase 2 clinical trial. This clinical trial is testing…
Gastric electrical stimulation (GES) has long been suggested as a treatment for symptoms of gastroparesis, but the use of the technique has been deemed controversial. A recent study published…
When you know your days are numbered, you might stop to think how you want to spend those last days. At times, medical emergencies can put patients into conditions or…
According to a story from Juvenile Arthritis News, the results of a recent phase 3 clinical trial indicate that the drug tofacitinib (marketed as Xeljanz) can be an effective treatment…
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…
According to a story from Neurology Advisor, the results of a recent study suggest that the presence of psychiatric issues in patients with idiopathic intracranial hypertension is connected to a…
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