Making meaningful contributions
PTC Therapeutics is committed to helping the lives of patients and families within our communities. Part of this commitment includes providing grants to support high quality programs that are focused on rare diseases and to make meaningful contributions globally.
All grant requests will be submitted to the PTC review committee for consideration and must have clear objectives and be in alignment with areas of interest to PTC. A complete grant proposal should be submitted 60 days prior to program start date.
We're here to help
We’re looking for ideas in motion. Initiatives poised to make a meaningful difference, while aligned with areas of focus at PTC.
Sponsorship and Investigator Initiated Study (IIS) requests follow separate PTC review processes. Refer to the drop downs below for more information:
Sponsorships are not part of the PTC Grant Program. If you are seeking financial support for a sponsorship, which is support for events or programs whereby PTC receives a tangible benefit (e.g. an annual membership in a professional society, an opportunity to set up an exhibit, display table or booth at the event, seats at a gala or a PTC speaking opportunity), the request is not a grant but is considered a sponsorship and is covered by a separate PTC procedure. Please contact your local PTC representative for submission of any sponsorship requests.
Investigator Initiated Studies are unsolicited studies in which the investigator conceives the research, develops the protocol, and serves as the sponsor. PTC provides support in the form of a medical research grant. All IIS requests will be submitted to the PTC review committee comprised of medical and scientific personnel for consideration. Independent requests must have clear objectives and be in alignment with areas of interest to PTC.
The committee evaluates the requests based on set criteria and evaluation process. A complete IIS proposal should be submitted 60 days prior to program start date.
For more information about Investigator Initiated Study (IIS) Requests, click here.
Medical Grants
PTC will consider funding for Medical Grants that fall under the following rare disease areas:
- Phenylketonuria (PKU)
- Familial chylomicronemia syndrome (FCS) (LATAM only)
- Familial partial lipodystrophy (FPL) (LATAM only)
- Hereditary transthyretin amyloidosis (hATTR) (LATAM only)
Last updated: February 2025
Patient Advocacy Grants
PTC will consider funding Patient Advocacy grants that fall under the following rare disease areas:
- Phenylketonuria (PKU)
- Familial chylomicronemia syndrome (FCS) (LATAM only)
- Familial partial lipodystrophy (FPL) (LATAM only)
- Hereditary transthyretin amyloidosis (hATTR) (LATAM only)
- Aromatic L-amino acid decarboxylase (AADC) deficiency
- Duchenne muscular dystrophy (DMD)
- Friedreich’s ataxia (FA)
Last updated: February 2025
Types of Grants
Grants to support scientific educational programs and events or other healthcare-related activities for Healthcare Professionals (HCPs). These activities are organized, controlled and conducted by an independent third party and include but are not limited to: continuing medical education (CME and non-CME), disease awareness, medical conferences, educational meetings, and medical initiatives that improve patient outcomes.
If related to an Investigator Initiated Study (IIS) request, click here for more information.
Contributions to support various projects, initiatives or non-profit organizations with bona-fide charitable missions with an affinity to PTC’s mission in communities where PTC has a presence (note: can include both healthcare-related and non-healthcare related charitable contributions).
Grants to provide funding of an individual (e.g., medical, pharmacy and/or nursing students, residents or fellows) to defray or cover reasonable costs of attending accredited continuing education (CE) activities (e.g., educational program, symposia, or conference) for academic or career advancement.
Grants to Patient Associations / Organizations to increase disease-state education/awareness or to provide support during and after diagnosis and/or treatment without PTC receiving any tangible benefit, such as; an annual membership in a professional society, an opportunity to set up an exhibit, display table or booth at the event, seats at a gala or a PTC speaking opportunity.
Grants to support activities regarding healthcare public policies and legislature.
A patient registry is an organized system to integrate all patients affected by a specific disease to collect health data and improve the outcome of the patients
In order to be considered for funding – not including IIS, please complete the below questionnaire to provide an initial understanding regarding the nature of your request:
Congratulations! Based on your responses, you are preliminarily qualified to submit a grant request for further consideration by PTC’s review committee.
Please provide the following information to register and receive credentials to access PTC’s Grants Portal. Once you are logged in to the portal, you will then be asked to complete a request form to capture additional details related to your grant request.
Already have log-in credentials to PTC’s Grants & Donations Portal? Click here to access.
Thank you for your interest in receiving funding support from PTC Therapeutics. Based on the information you have provided, regrettably we are unable to provide support on this occasion.
We receive many worthwhile requests each year, but unfortunately, it is not always possible to support each request. Please contact us if you have any queries, or if you need any further information.
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.