Interim results a from Phase 1/2 clinical trial support the therapeutic benefits of investigational therapy combination ATB200/AT2221 in patients with Pompe disease. Participants in the study showed improvements in motor and respiratory function. The therapy also resulted in a durable decrease of muscle damage biomarkers. These results will be presented…
treatment
The biotech companies Shire and NanoMedSyn have launched a new research partnership to evaluate a potential treatment for lysosomal storage disorders including Pompe disease. NanoMedSyn has developed an enzyme replacement therapy using proprietary technology called AMFA that was shown in preclinical models to have promising biological activity.
Mitochondrial disorders are complex conditions for which few treatments are available. Now, using microscopic worms, researchers at The Children’s Hospital of Philadelphia (CHOP), are researching if existing human drugs aimed at improving metabolism and restore shortened lifespans, can open the opportunity for human clinical trials on innovative therapies for these…
Recent Posts
- Home enzyme replacement therapy safe for Pompe disease children
- What ‘lucky’ really means for a child with Pompe disease
- Infantile Pompe brain changes may begin long before signs appear
- 1-time gene therapy cuts need for enzyme treatments for Pompe children
- What a child with Pompe disease taught me about being a parent