FDA Patient Listening Sessions
Help the FDA better understand Proteus Syndrome.
December 1, 2022, from 12 – 1:30 PM ET
The U.S. FDA’s Office of Patient Affairs, in collaboration with National Organization for Rare Disorders (NORD), is organizing a virtual Patient Listening Session on Proteus Syndrome. The FDA would like to better understand how this disorder and its symptoms impact people living with proteus syndrome, how they manage their symptoms, and their perspectives on clinical trials. Please download the file below to learn more.
December 1, 2022, from 12 – 1:30 PM ET
The U.S. FDA’s Office of Patient Affairs, in collaboration with National Organization for Rare Disorders (NORD), is organizing a virtual Patient Listening Session on Proteus Syndrome. The FDA would like to better understand how this disorder and its symptoms impact people living with proteus syndrome, how they manage their symptoms, and their perspectives on clinical trials. Please download the file below to learn more.
| flyer_for_proteus_family_conference.pdf |
AIM Inhibitor Miransertib in Proteus Syndrome Trial - Trial start Summer 2022.
Phase 2 of the clinical trials will begin this summer at the National Institutes of Health in Bethesda, MD. For more information please call: Call or Email.
Proteus Syndrome Foundation
Welcome to the Proteus Syndrome Foundation (PSF). We are composed of families who connect with each other through the foundation and professionals who work tirelessly to help find effective treatments for Proteus. The PSF provides support through family conferences, family sharing through our (new) PSF Forum on our web page, and grants for families in need of assistance. Proteus syndrome can be an isolating and scary syndrome but as we come together under the PSF umbrella, we will never stand alone.
What is Proteus Syndrome?
Proteus Syndrome is a condition which involves atypical growth of the bones, skin and head, and can lead to a variety of other symptoms. The condition is caused by a genetic mutation in AKT1, an important gene that helps to regulate the growth of cells. Now that genetic testing for the AKT1 mutation is available, it is important to get tested. In the spring of 2019 the PSF will be able to offer this confidential testing to families.
Please contact the Proteus Syndrome Foundation to learn more.
Please contact the Proteus Syndrome Foundation to learn more.
PSF Family ConferenceThe next PSF Family Conference will be held on October 7 & 8, 2022 in Bethesda Md. Registration and more information coming soon.
Watch the video of Dr. Leslie Biesecker’s 2017 Proteus Syndrome Family Conference presentation on ARQ092:
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Patient Number FivePatient Number Five is a documentary that follows people diagnosed with Proteus Syndrome as they enter clinical trials for a new drug miransertib (formerly called ARQ092). This new drug could be the first approved treatment that patients in critical condition desperately need.
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Contact RegistryThe Proteus Syndrome Foundation Contact Registry will be used to inform individuals with Proteus syndrome and their guardians about:
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Proteus Syndrome Foundation - 9296 Penobscot Ct., Colorado Springs, CO. 80924 USA
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