Gene Therapy improved vision in patients with leber hereditary optic neuropathy (LHON) By: Dr. Maria Niu Mitochondria, powerhouses of eukaryotic cells, is a certain kind of cytoplasmic organelle and plays a critical role in energy production. Mitochondria dysfunction results in a broad spectrum of multisystem disorders. LHON is a mitochondrial neurodegenerative disease typically caused by […]
Biologic treatments show promise in providing clinical solutions to a variety of diseases including rare cancers and precision medicine. Services include: Indications analysis and prioritization Strategic drug assessment Clinical study design and protocol Biomarker strategy Early Clinical Development FDA meeting and submission Pre-ND integrated development plan CRO and project management Study remediation and rescue […]
Biomarkers are now a routine part of drug development The FDA recognizes biomarker development as a high priority area for future research. The FDA and European Medicines Agency (EMA) have developed similar processes for the qualification of biomarkers intended for use as companion diagnostics or for development and regulatory approval of a drug or therapeutic. […]
The World Orphan Drug Congress USA will be a live virtual conference. BBCR’s Team invites you to send an email at [email protected]/bbcr2. We hope to meet many of you that will attend the World Orphan Drug Congress. We are very much interested in learning about your programs to determine if BBCR’s expertise in Rare Diseases […]