Improving outcomes through collaboration and partnership
A hub for the latest medical information on rare disease
- Early and accurate diagnosis and management of rare disease
- Best practice information
- Abstracts, posters, webinars, and presentations from medical congresses
- The latest scientific data and expert insights on rare disease across neuromuscular, CNS, and more
A sincere passion for patients. A deep roster of talent with unmatched expertise. Strong technology platforms. These elements combine to supercharge our robust discovery pipeline for patients living with rare disorders.
Make an investigator-initiated study (IIS) request
IIS requests are unsolicited studies where the investigator conceives the research, develops the protocol, and serves as the sponsor. Independent requests must have clear objectives and be in alignment with PTC’s mission. A complete IIS proposal should be submitted 60 days prior to program start date.