The New England Journal of Medicine recently carried a report citing data from a Phase 3 clinical trial involving 307 people diagnosed with MSI-H-dMMR metastatic colorectal cancer. MSI-H (high levels…
The Yale Cancer Center has recently presented the findings of their study of the cost-effectiveness of caplacizumab at the 62nc ASH Annual Meeting and Exposition. Their conclusion, which is that…
On November 27, biotechnology company Vertex Pharmaceuticals Inc. ("Vertex") announced the approval of SYMKEVI with KALYDECO for the treatment of pediatric patients with cystic fibrosis (CF), says GuruFocus. The…
by Lauren Taylor from In The Cloud Copy Pantothenate kinase-associated neurodegeneration or PKAN is a rare, inherited condition in which the affected child experiences progressive degeneration in certain parts of…
As of November 20, Zokinvy has been approved by the FDA for the treatment of Hutchinson-Gilford progeria syndrome (progeria) and a few other progeroid laminopathies in patients aged one year…
Bulgarian cystic fibrosis patient joins international campaign standing up for the rights of patients to access essential medicines Rositsa Malinova, a 22-year-old resident of Pazardzhik, Bulgaria, is taking on the…
From December 1-4, 2020, Reuters hosted a virtual conference called Pharma & Patient USA 2020. The primary focus of this event was furthering patient centricity in disease development as well…
In a press release from late November, pharmaceutical company BioCryst Pharmaceuticals, Inc. ("BioCryst") announced the publication of data from its APeX-J clinical trial in Allergy. During the trial, which was…
A study in the European Medical Journal aims to confirm the safety profile of vedolizumab, a treatment for inflammatory bowel disease (IBD). Specifically, they focused on acute infusion reactions. It…
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,…
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