Thank you for your generous donation, which will be used to help find treatments for Nay and other kids with ML4!

Your gift supports research at Massachusetts General Hospital/Harvard where we are creating a gene therapy for Mucolipidosis Type IV and developing a natural history (a study of all the patients in the world) of MLIV. This incredible treatment inserts a functioning gene currently missing in people with MLIV, and the natural history builds a detailed medical picture of this disease which is required for successful treatment. Your support directly helps fund the scientists creating this treatment and the doctor who will treat these children. Even during COVID, these researchers are pushing forward with this life-changing work.

Your generosity makes this work possible. We could not succeed without you.

Again, thank you for helping us reach our goals! Please stay connected with us at www.ML4.org.

This notice provides official acknowledgment of your donation to The Mucolipidosis Type IV (ML4) Foundation. No goods or services were provided in exchange for this contribution. The ML4 Foundation is an U.S. exempt organization as described in Section 501(c)3 of the Internal Revenue Code; (EIN 13-3633501). If we have your valid mailing address, you will also receive an acknowledgment by mail.

Hope for Nay

Raising funds to find treatments for ML4

Team Members