As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has granted priority review to BridgeBio Pharma’s investigational therapy, BBP-418, marking a key regulatory
Formally known as Ebola hemorrhagic fever, Ebola is often fatal in humans. The disease is transmitted through contact made with body fluids or blood of
Editor’s Note: This article was originally written and shared with us by Caleigh Haber. This day hits really close to home for me. I was
As reported by PharmaBiz, Japan’s Ministry of Health, Labour and Welfare (MHLW) has broadened the approved use of GSK’s respiratory syncytial virus (RSV) vaccine, Arexvy,
According to a recent article on PharmaBiz, HAYA Therapeutics has announced the completion of dosing in the first cohort of its phase 1 clinical study
According to a recent article in The Manila Times, Jade Biosciences has begun dosing participants in a Phase 2 clinical study, known as JUNIPER, evaluating
An article from Medscape highlighted recent advances in treatment that are reshaping the clinical approach to epidermolysis bullosa (EB), transitioning care from largely supportive management
A recent article by BioPharmaDive highlighted a mid-stage clinical trial that evaluted an investigational Parkinson’s disease therapy co-developed by Biogen and Denali Therapeutics, and that
Johnson & Johnson announced FDA approval of a supplemental new drug application for Caplyta (lumateperone) for the prevention of relapse in schizophrenia, based on compelling
A growing body of research highlights the broader benefits of human papillomavirus (HPV) vaccination beyond its long-standing focus on cervical cancer prevention. A recent large-scale
According to a recent article by The Manila Times, Design Therapeutics has reported encouraging interim results from its ongoing Phase 1/2 RESTORE-FA study, highlighting both
Novartis and PTC Therapeutics have advanced their Huntington’s disease candidate, votoplam, into Phase 3 development following promising mid-stage results. The decision reflects confidence in the
As reported on MedicalXpress, a newly developed RNA-focused analytical method may improve the detection and characterization of repeat expansion disorders, a group of genetic conditions
A recent article from The Manila Times highlighted findings from the ongoing IMPACT‑TD Registry underscore a persistent gap in diagnosing tardive dyskinesia (TD), particularly among
BridgeBio’s Attruby (acoramidis) is positioning itself as a formidable competitor in the transthyretin amyloid cardiomyopathy (ATTR-CM) market, with new pivotal trial data and indirect comparisons
A recent article from SciTechDaily describes the promising approach instituted at a private, non-profit biomedical research firm in San Francisco that incorporates data science, AI,
According to a recent press release, Artios Pharma has begun dosing patients in a global Phase 2 clinical trial evaluating its investigational DNA polymerase theta
A significant advancement in antiviral therapy has emerged with the successful development of a CRISPR-Cas13d-based system designed to combat hepatitis E virus (HEV) infections. This
Cystinosis Research NetworkCystinosis is a rare, genetic, metabolic, lysosomal storage disease that causes an abnormal accumulation of the amino acid cystine in various organs and tissues of the body.
For more info, visit our website (linked in comments)
#cystinosis #RareEdu #CystinosisAware
Jordan's Guardian Angels Northern Ireland Rare Disease PartnershipIrelandRareDiseasePartnership
The Life Raft Group #GistStay up to date on the latest #GISTresearch presented at ASCO 2026! Join Dr. Hirotaka Miyashita of UC San Diego Health on June 17 as he reviews key abstracts and presentations relevant to the #GISTcommunity.
Register today: bit.ly/UpdatesASCO2026
#GIST #ASCO26 #CancerResearch
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