Atara Therapeutics has released data from its Phase 1 open-label extension (OLE) trial of ATA188, a treatment for progressive multiple sclerosis (MS). This allogeneic T-cell immunotherapy targets B cells and…
Previously, clinical-stage genetic medicines company Homology Medicines, Inc. ("Homology") submitted an Investigational New Drug (IND) application to the FDA for HMI-103, an investigational gene editing therapy designed to treat phenylketonuria…
The 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) was held last week from the 13th to the 15th. This event is the largest…
According to The Guardian, England’s National Health Service (NHS) has announced that a new therapeutic option will be available for patients with sickle cell disease (SCD). This treatment, called crizanlizumab,…
In June 2021, Redx Pharma dosed its first healthy volunteer in a Phase 1 clinical trial evaluating the safety and phamacological profile of RXC007. Altogether, this investigational therapy is designed…
According to a news release in Yahoo! Finance, biopharmaceutical company Zogenix, Inc. recently submitted a Supplemental New Drug Application (sNDA) for its drug candidate FINTEPLA (fenfluramine). The therapy is designed…
According to a recent article from UTHealth Houston, a five-year-old suffering from a severe case of spina bifida has brought together a number of doctors to give her the…
At two years old, Charlotte Wilson, known affectionately as Lottie, has such a bright and happy smile. Her family, including her mother Lisa, feels lucky to still have her…
In a news release from September 30, 2021, biopharmaceutical company Arrowhead Pharmaceuticals ("Arrowhead") shared that it had initiated its Phase 2b AROAPOC3-2002 clinical trial and that the first patient…
Dr. Ed Neilan, who is the Chief Medical and Scientific Officer of the National Organization for Rare Disorders (NORD), recently commented that there are approximately more than seven thousand…
Technology can assist sponsors in setting up trials and activating clinical trial sites quickly. But tech will only play a small role within the larger movement toward accelerated approval. Read…
Fast Track designation is requested by the drug company; this process and designation are designed to facilitate the development and review of therapies which can treat serious or underserved conditions,…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In the past, imatinib has shown promise for treating patients with pulmonary arterial hypertension (PAH). But specialty pharmaceutical company Tenax Therapeutics, Inc. ("Tenax") believed that it could create a specialty,…
Written by Robyn Stacy-Humphries Wolverine, Spiderman, Wonder Woman, and Deadpool - superheroes most of us love. Why? They have superpowers and escape death by some type of genetic alteration. Humans…
Author Lauren Wellbank recently interviewed Dr. Sara Candler, Director of the Iora Care Team, for a special edition of Martha Stewart’s Health and Wellness. The interview focused on dementia and…
October 23 is recognized each year as Kabuki Syndrome Awareness Day, a time to help spread awareness about this syndrome among the general public and in the medical field. Kabuki…
Clinical trials can be helpful tools in determining the safety, efficacy, tolerability, recommended dose, and pharmacokinetics of specific drugs (among other things). In doing so, these trials help tell researchers…
Now that it's officially October, it's time to celebrate my personal favorite holiday - Halloween. That means haunted houses, scary movies, creative costumes, and spooky decorations. One Transcona family is…
Silica is a naturally occurring substance found in various types of rocks, clay, and sand. Unfortunately, when this silica dust is inhaled, especially over long periods of time, it can…
Two and a half years after announcing its launch, Dyne Therapeutics held its very first Research & Development Day. Throughout the day, there were presentations focusing on the development of…
Did you know—October is known as Spina Bifida Awareness Month, with October 25 being designated as World Spina Bifida and Hydrocephalus Day? That means, all October long, you can help…
Written by Lori Lawter, MPH We’ve heard a lot about bringing drugs to market lately, especially in light of the COVID-19 vaccine. You might wonder, “Is there a ‘secret sauce’…
Sometimes, companies work together to develop the best therapeutic options for patients. According to Optical Prism, biopharmaceutical company reVision Therapeutics, Inc. and specialty pharmaceutical company Aequus Pharmaceuticals Inc. recently joined…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
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