Every clinical study has its unique challenges that initially may not have been factored for. Experienced management can help sponsors to address prolonged trial timeline and high quality data. Rare Diseases and Precision Medicine Require Unique Approaches In Clinical Trial Design. There could be any number of factors that could necessitate a study being rescued. […]
Retrophin’s experimental PKAN drug fosmetpantotenate fails phase III trial Pantothenate kinase-associated neurodegeneration (PKAN) is a rare disease characterized by a progressive neurodegenerative disorder and buildup of iron in the brain which is estimated to affect up to 5000 patients worldwide. PKAN is inherited in an autosomal recessive manner and is caused by mutations in the […]
Neuroinflammation is inflammation of the nervous tissue. It may be initiated in response to a variety of cues, including infection, traumatic brain injury, toxic metabolites, or autoimmunity. In the CNS, including the brain and spinal cord, microglia are the resident innate immune cells that are activated in response to these cues. The CNS is typically an immunologically privileged site because peripheral […]
In practice, for a given clinical trial, it is not uncommon to have 3–5 protocol amendments after the initiation of the clinical trial. One of the major impacts of many protocol amendments is that the target patient population may have been shifted during the process, which may have resulted in a totally different target patient […]
BBCR Consulting, for the first time this year, decided to attend on October 15th – 17th in London- UK, the European edition of the Gene Therapies in Rare Disorders conference. In our opinion, the event, as promised, proven to be: ”…uniquely focused conference that will bring the leaders in the field together to discuss the […]
Biologic treatments show promise in providing clinical solutions to a variety of diseases Due to biological’s unique characteristics, study designers may find that results in pre-clinical trials do not predict acute, chronic and late onset immunogenicity and safety. In Addition, the biological orphan has a long PK half-life of months such as with any mAb, […]
People tend to believe that a repurposed therapy can never be truly novel or transformative. Nothing could be further from the truth. One attractive option of Drug Repurposing is to use a scientific approach to identify new uses for existing drugs. About a third of orphan approvals by the FDA since the program began have […]
