In 2015, the United States approved Kanuma (sebelipase alfa) for the treatment of infants living with rare lysosomal acid lipase deficiency. However, the therapy was not approved in other countries;…
According to a story from Healio, a recent study has found that use of hydroxyurea in alignment with treatment guidelines has the potential to bring down symptoms of cerebrovascular disease…
According to a story from Healio, Rohit Kohli, MBBS, MS, division chief of gastroenterology, hepatology and nutrition at Children’s Hospital Los Angeles, spoke at the World Congress on Insulin Resistance, Diabetes…
The drug Aficamten is being developed by Cytokinetics as a pill taken once daily for hypertrophic cardiomyopathy (HCM). It is a genetic disease where due to a thickness in the…
In 2023, there have been new drug approvals and increased patient awareness. The American Cancer Society reported a total of 1,958,310 new cancer cases in 2023. Chemotherapy remains the standard…
Carisma Therapeutics, the manufacturer of CT-0508, issued a press release stating that researchers are conducting a combination study using CAR-M cell therapy plus Keytruda (pembrolizumab) in the treatment of overexpressing…
According to a story from Science Daily, a team of scientists affiliated with the Salk Institute recently published a study on December 6, 2023 focused on how a class of…
According to a recent American Society of Clinical Oncology (ASCO) press release, Dr. Jason Westin provided testimony regarding a nationwide shortage of cancer drugs, numbering more than 15 on the…
The treatment of young patients with R/R acute myeloid leukemia (AML) poses a challenge for clinicians. Chemotherapy regimens, such as high doses of cytarabine, fludarabine, and other salvage reduction therapies,…
According to a story from BioPharma Reporter, the pharmaceutical company Candel Therapeutics recently announced that its investigational therapy designated CAN-2409 has earned Fast Track designation from the US Food and…
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…
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…
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