According to a story from Hematology Advisor, a recent study released in Scientific Reports indicates that gene expression has the potential to predict both primary resistance and treatment response in patients living…
According to a story from GlobeNewswire, the biotechnology company Galecto, Inc., has recently published the results from its phase 2a clinical trial. This study was testing the company's experimental therapy…
Mission: Cure is a coalition of patients, researchers, doctors, and entrepreneurs who have taken a new approach to curing diseases. Their first focus is pancreatitis, and they have created a…
Recently, biopharmaceutical company Acceleron Pharma Inc. ("Acceleron") announced that its investigational therapy ACE-1334 was granted Orphan Drug designation by the FDA. The treatment is designed for patients with systemic…
Part of the National Institutes of Health (NIH), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and SteroTherapeutics have entered into a Cooperative Research and…
Transitions to adult care for patients who have a chronic respiratory disease, like cystic fibrosis, or a type of neuromuscular disease that influences the respiratory system is something that the medical…
The FDA has recently approved DANYELZA for the treatment of neuroblastoma in pediatric patients over the age of one and adult patients affected by refractory or relapsed neuroblastoma. This approval…
According to a press release from Inovio Pharmaceuticals, the biotechnology company has officially dosed the first patient in its phase 1/2 clinical trial, which will test the company's investigational therapeutic…
WCVB NewsCenter 5 recently published a news item featuring Nancy and Paul Burke and their three daughters. The Burke family has been living in the shadows of the deadly Sanfilippo…
An article recently appeared in Health News by NPR describing progeria, an extremely rare disease. There are only twenty people in the United States diagnosed with progeria. The disease causes…
Sorreto Therapeutics recently announced through Globe Newswire that it has filed an application for its investigational new drug COVI-DROPS (STI-2099). The study will investigate the safety and efficacy of the…
There is a stigma that comes with the term "palliative care," as it is typically associated with hospice and terminal illnesses. This is not the case, as palliative care is…
Maintaining lung function is a major goal of cystic fibrosis (CF) treatment. In order to accomplish this goal, doctors will use airway clearance techniques, such as vest therapy. The problem…
COVID-19’s tentacles have invaded the New Drug Applications (NDAs) for 2020. A recent article in Fierce Pharma reported that the FDA has been especially vigilant in scrutinizing NDAs this year,…
This week Hallmark Movies and Mysteries premiered The Christmas Bow, which features Joy Perry, a woman with Charcot-Marie-Tooth disease, playing a woman with CMT. And while CMT has been known…
Innovent Biologics has recently received approval from the National Medical Products Administration (NMPA) for their juvenile idiopathic arthritis (JIA) treatment, SULINNO. It has been approved to treat JIA, rheumatoid arthritis,…
The latest news from Israeli scientists, published in the Times of Israel, is that researchers at Tel Aviv University have accurately targeted cancerous cells in mice using CRISPR.…
On November 19, 2020, Research!America hosted an informational webinar titled "From Nobel Winning Science to Next Generation Treatment: Tracing the Path of a Rare Disease Breakthrough using RNAi." This webinar focused…
In a recent press release, gene therapy company Orchard Therapeutics ("Orchard") announced that the FDA approved Orchard's Investigational New Drug (IND) application for OTL-200. This gene therapy solution is…
According to a report in BioPharma Dive, Brainstorm Therapeutics, a New York biotechnology company, said that while its NurOwn therapy for amyotrophic lateral sclerosis (ALS) appeared to have a…
From November 13-16, researchers, doctors, and others participated in the American Association for the Study of Liver Diseases (AASLD)'s Annual Meeting, or The Liver Meeting Digital Experience. During the…
In late November, RNAi therapeutics company Alnylam Pharmaceuticals ("Alnylam") announced that its therapy OXLUMO (Lumasiran) received approval from the European Commission (EC) for both pediatric and adult patients with…
When gene therapy company Asklepios BioPharmaceutical, Inc. ("AskBio") and biotechnology company Selecta Biosciences ("Selecta") joined forces to combat genetic disorders, each company brought its best technology and insights. AskBio…
Takeda Pharmaceuticals has recently awarded XOMA Corporation $2 million for reaching the next milestone in the development of mezagitamab, a treatment for myasthenia gravis (MG). According to Biospace, XOMA has…
According to a press release from the biopharmaceutical company Sanofi, the company's investigational treatment avalglucosidase alfa will have its Biologics License Application (BLA) evaluated by the US Food and Drug…
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