April 15, 2020

Today is our 7^th International Pompe Day!

The goal of International Pompe Day is to foster international awareness of Pompe Disease and the slogan for our day is Together We Are Strong.

The IPA believes that raising awareness about Pompe Disease is key, and the theme selected for 2020 is Hope For The Future: It is an exciting time for the Pompe Community worldwide with new studies and treatments on the horizon, and Hope is what drives the patient community, and what inspires the medical\scientific community, and industry, to keep working.

And while the current Covid-19 pandemic is unprecedented, and we are trying to take each day as it comes due to the uncertainty, now, more than ever, we need to have hope and spread hope, we will get through together as a united community and something positive will come out of it.

Our Hopes For the Future have been collected in a dedicated blog that the IPA has specifically created: ipompeday.blogspot.com/2020

Thanks to all that have contributed, and happy International Pompe Day to our amazing Pompe community, to the Pompe warriors, families, carers, medical specialists and all those collecting our hopes and contributing to a better future!

Courtesy of the AMDA - Acid Maltase Deficiency Association.

With all of the questions and uncertainty surrounding the ongoing COVID-19 pandemic, the AMDA worked with Dr. Priya Kishnani and her group at Duke University on the following Question and Answer Fact Sheet. We hope that the Pompe Community will find the information included helpful.

Please note that this was based on information available as of 3/19/20. This is a constantly evolving situation. As more information becomes available, we will strive to promptly update this information as necessary.

To read the AMDA Question & Answer Fact Sheet, please click here.

Disclaimer: the current situation regarding the COVID-19 health emergency varies country by country and therefore some of the information may not be relevant to your country-specific situation. In the event of questions and/or uncertainties, the IPA encourages to reach out to national patient organizations and treating physicians.

Dear IPA Board of Directors,
As I’m sure you are aware, the World Health Organization (WHO) has declared a pandemic in response to the COVID-19 (SARS-CoV-2) outbreak. Given this serious health situation, the US Centers for Disease Control and Prevention (CDC) guidelines consider individuals with certain serious chronic medical conditions an especially at-risk population for more severe cases of COVID-19 (SARS-CoV-2). 
After consultation with global investigators, advocacy leadership, and healthcare experts, and in an abundance of caution for the health and safety of people with Pompe disease, Spark Therapeutics has decided to voluntarily suspend enrollment into SPK-GAA-100 (seroprevalence study) and RESOLUTE (SPK-GAA-101, the Phase I/II gene transfer study) at this time.  This voluntary suspension is solely related to the COVID-19 (SARS-CoV-2) pandemic and by suspending enrollment we aim to minimize risk of exposure to COVID-19 (SARS-CoV-2) for participants who would have been traveling to and from investigational sites. 
Please note that the recommendation to suspend enrollment in RESOLUTE (SPK-3006-101) is only currently applicable to investigative sites in the US, as EU sites are not yet open to enrolling.  Pre-screening activities that can be handled via email or phone may continue, and remote work to ensure that sites in both the US and Europe are positioned to enroll as soon as possible is continuing.  As the situation with COVID-19 (SARS-CoV-2) evolves, we will be in close communication regarding this voluntary suspension, especially when we have plans to resume enrollment.
As a leader in the field of gene therapy, we have always placed patient safety first and foremost in our research.  During this unprecedented time, we feel this is the responsible way to safeguard the well-being of individuals with Pompe disease and their caregivers who were planning to join our Pompe clinical research programs.  Spark Therapeutics remains committed to investigating a potential gene therapy for Pompe disease and restarting the SPK-GAA-100 study and RESOLUTE (SPK-GAA-101) as soon as safely possible for participants. 
Please let me know if you have any questions.  I am happy to set up a call at your convenience if you would like to discuss further.  Thank you for your continued collaboration on our shared goal of supporting the Pompe community. 

Take care, 
Amy K. Fisher, MS, CGC
Patient Advocacy Lead
Spark Therapeutics

Disclaimer: The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.