First Two Children Receive New Gene Therapy for Rett Syndrome
Neurogene Inc.
Phase 1/2 trial showed the safety and therapeutic potential of administering a novel transgene
Therapeutics
Targeting Viral Protein May Reduce HIV-Related Comorbidities
University of Montreal Hospital Research Centre
Per the WHO records, more than 39 million people were living with HIV at the end of 2022
BCG Challenge Unlocks Insights into TB Vaccine Research
Saint Louis University
Scientists believe they can use one vaccine to study the modus operandi and efficacy of others
FDA Approves First Drug Therapy for Desmoid Tumors
U.S. Food and Drug Administration
The FDA granted the approval of Ogsiveo to SpringWorks Therapeutics Inc.
Gene Therapy to Treat Rare and Hereditary Diseases
Norwegian University of Science And Technology
Researchers are finding clues that may help treat rare and deadly diseases using mini-organs and advanced technology
Combination Therapy Improves Rare Brain Tumors in Children
Great Ormond Street Hospital
Trials showed the effect of combination therapy on survival and quality of life in children with low- and high-grade gliomas
New Approach to Using Immunotherapy Combinations in Bladder Cancer
Netherlands Cancer Institute
Nivolumab and gemcitabine–cisplatin combo showed significantly better outcomes than standard-of-care in patients with advanced urothelial carcinoma
Trials Reveal First-Ever MDR-TB Treatment Safe for Kids and Adults
Desmond Tutu TB Centre at Stellenbosch University
Bayesian analysis of data from both trials showed that levofloxacin reduced the risk of developing TB by 60 percent
RNA Expression Scores Predict Response to Bladder Cancer Therapy
SWOG Cancer Research Network
Prospective trials may help assess whether molecular subtype-directed approach can improve response to neoadjuvant therapy in MIBC patients
AI’s Impact on Drug Development: Opportunities for Pharmaceutical Leaders
Claire Aldridge, PhD
Explore the transformative impact of artificial intelligence on drug development and its role in the future of pharmaceutical innovation
FDA Approves First Vaccine for Chikungunya Disease
U.S. Food and Drug Administration
Ixchiq is a single-dose, attenuated vaccine, safe to be administered to individuals above 18 years
FDA Approves Recombinant Enzyme to Treat Rare Blood Clotting Disorder
U.S. Food and Drug Administration
Adzynma safely and effectively “filled in” for the mutated ADAMTS13 enzyme in patients with cTTP
Gender-Neutral Vaccination Prevents Oncogenic HPV Most Effectively
Karolinska Institutet
Vaccinating boys and girls induces herd immunity that helps eradicate oncogenic HPV types more quickly
Adjuvanted Recombinant COVID-19 Vaccine Outperforms Competitors
Korea University
The GB510/AS03 vaccine elicits a stronger antibody immune response against the ancestral SARS-CoV-2 strain than ChAdOx1-S
Combo Immunotherapy Trial for Glioblastoma Commences
University of North Carolina School of Medicine
New Drug for Relapsed Blood Cancer in First Human Trial
The James Cancer Hospital and Solove Research Institute (OSUCCC – James)
CLL makes up a quarter of leukemia cases among adults and NHL accounts for 4 percent of all cancers in the US
Novel Drug Improves Rare Kidney Disorder in Phase 3 Trial
University of Minnesota Medical School
Some 42 percent of patients treated with sparsentan showed reduced proteinuria
FDA Approves Interchangeable Biosimilar for Multiple Inflammatory Diseases
U.S. Food and Drug Administration
Wezlana has similar safety and efficacy as its reference product and is a cost-effective alternative for chronic inflammation
Progress in Precision Oncology: AACR Cancer Progress Report 2023
Michael Schubert, PhD
Liquid biopsy, molecular profiling, and other recent advances from the AACR Cancer Progress Report 2023
Antibody-Drug Conjugate Benefits HER2-Low Breast Cancers
German Institute for Quality and Efficiency in Health Care (IQWiG)
Patients with HER2-low breast cancers were included in the clinical trial proving the efficacy of trastuzumab deruxtecan monotherapy