Tagged: rare disease

PKAN drug fails Phase III trial: How to avoid a similar disaster.

November 26th, 2019 | rare disease

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 […]

Biologic treatments show promise in providing clinical solutions to a variety of diseases. Boston Biotech Clinical Research has experience in biologics for rare diseases and can help develop a targeted strategy for your needs.

October 31st, 2019 | rare disease

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, […]