A recent release published in BioSpace announced that a gene therapy company is vigorously pursuing the study of AVB-101 to treat patients with frontotemporal dementia (FTD), including FTD with GRN…
GRIN2B Disorders are rare changes in the GRIN2B gene, some causing gain of function, some loss of function, and others unusual neurotransmissions, but all resulting in a variety, and a…
The increasing penetration of digital technologies and the ubiquity of smartphones and the internet have transformed several spheres of our lives. Healthcare is no exception. With technological advances, it has…
In 2013, the Morells welcomed two identical baby girls, Kaylie and Kenzie. The joy they felt soon disappeared as the twins began to slip back from their normal infant and…
Bladder cancer patients and their families have faced years, and even decades, of waiting for a breakthrough in bladder cancer treatment. For some patients, the results of two clinical…
A post hoc analysis of CIMZIA (certolizumab pegol) compared to the drug adalimumab for treating rheumatoid arthritis (RA) patients was presented at the November 2023 ACR Convergence in San Diego.…
According to a story from drugs.com, a combination therapy for chronic kidney disease has shown considerable potential in a recent phase IIb clinical trial. The combination being evaluated consisted of…
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell disorder in which blood cells break apart prematurely. In an effort to bring more effective and targeted treatments…
Neuroblastoma most commonly occurs in children under the age of five. The disease usually affects areas of the adrenal gland (positioned over the kidneys) or areas in the neck, chest,…
Roche has added another failure this year with its drug gantenerumab. As noted in Fierce Biotech, the drug was unable to improve functional or cognitive decline in two Phase…
Two emergency surgeries that began 48 hours after birth, meningitis, and over a month in the intensive care units of three Seattle hospitals prompted Layla Babayev’s desperate parents to enroll…
The biotechnology company Cabaletta Bio has recently announced that the company investigational new drug (IND) application for its experimental CD19-CAR T-cell therapy CABA-201 has been cleared by the US Food…
Prior to the FDA’s approval this month, nirogacestat (Ogsiveo) received the following FDA designations applicable to the treatment of desmoid tumors: Breakthrough therapy providing a rapid review. Fast track to…
Meenakshi Rao, an assistant pediatric professor at the Children’s Hospital in Boston, recently expressed his opinion to the RNA-Seq blog that inflammation, not bacteria, should be targeted, as infections…
Written by Maria Palombini Answer: at the time of diagnosis, it should be one of the first actions you, the patient and/or caregiver, should take. Why? Receiving a diagnosis with…
According to a story from BioPharma Dive, the pharmaceutical company Celldex Therapeutics recently released positive results from two clinical trials. These trials involved the company's investigational therapy, barzolvolimab. In a…
Clinical studies are critical tools in identifying and developing therapeutics for a wide variety of conditions. This research is especially important in the rare disease and chronic illness spaces,…
The goal of providing precision techniques to cancer care is becoming increasingly complicated. According to a recent article in Inside Precision Medicine, the cause may be attributed to the…
Historically, drug development within the rare disease space has been stifled; companies tend to focus on developing therapies for bigger patient populations that will be more impactful and more…
CRISPR was first described ten years ago as a viral mechanism for defense, taken from bacteria, which could be transformed into genetic scissors and accurately alter DNA. About Casgevy CRISPR…
According to a story from Pharmaceutical Business Review, the UK's National Institute for Health and Care Excellence (NICE) is backing daridorexant (marketed as QUVIVIQ) as a treatment for chronic insomnia…
Patients with severe asthma with an eosinophilic phenotype who are 12 years of age or older may now receive treatment with FASENRA as an add-on maintenance treatment. Note that…
J.D. Martinez is a designated hitter and outfielder for the LA Dodgers. According to a story from The Santa Clarita Valley Signal, he also lives with asthma, which can have…
Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side…
In 2005, the U.S. Food and Drug Administration (FDA) approved abatacept for the treatment of moderate-to-severe rheumatoid arthritis. Twelve years later, in 2017, the FDA expanded the drug's approval…
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