Wyeth-Ayerst sites for myostatin inhibitor MYO-029 clinical trial on facioscapulohumeral muscular dystrophy (FSHD) are listed below.

This is for your information.  Get the word out to your family members with facioscapulohumeral muscular dystrophy (FSHD) and those you know who live close to the clinical trial test sites.  Contact the doctors below if you are interested in participating in the trials.

Myostatin inhibitors have never been tried in human trials.  This is the first drug and first trial.  FSHD is the lead candidate and prospect thanks to the work of the FSH Society, Inc.

Each site will enroll 12 patients.  4 FSHD, 4 LGMD, 4 Becker MD.

Initial toxicity studies are done.  There some side effects that are consistent with monoclonal antibody therapy.

This next stage of the trial is to determine dosing at three different levels and clinical efficacy.

Patients must be genetically confirmed.

The MSTN-inhibitor drug is administered via intravenous infusion.

Initial patient visit/transfusion may or may nor require an overnight stay.  New infusion every other week for six months and three more months follow-up.  Two muscle biopsies are optional. 

Patients should have average muscle strength grade of 3.  As general rule patient should be able to walk 30 feet unaided except for use of orthotic braces e.g. AFOs.

9 sites U.S.. 2 or 3 sites U.K. are currently enrolling four FSHD patients and you may wish to contact the following.  Most will request that you be within one hour of the hospital given the number of visits.

Wyeth-Ayerst sites for myostatin inhibitor MYO-029 clinical trial:

List based on my best information to date.

Brigham and Women’s Hospital, Boston (Anthony Amato)

University of Rochester, Rochester, New York (Rabi Tawil)

Washington University, St. Louis, Missouri (Alan Pestronk)

Johns Hopkin’s Medical, Baltimore, Maryland (Kathryn Wagner)

Kansas University Medical Center, Kansas City, Kansas (David Saperstein)

University of Utah, Salt Lake City, Utah (Kevin Flanigan)

Children’s Hospital DC, Washington DC (Diana Escolar)

Ohio State University, Columbus, Ohio (Jerry Mendell)

University of Texas Southwestern, Dallas, Texas (Gil Wolfe)

University of Newcastle upon Tyne, Newcastle upon Tyne, United Kingdom (Kate Bushby)

King's College Hospital, London, England (Michael Rose, Caroline Murphy)

I am uncertain about Oxford.

Oxford University, Oxford, United Kingdom (David Hilton-Jones)

My best,

Daniel Paul Perez

President & CEO

FSH Society, Inc.

FSH Society, Inc.

3 Westwood Road

Lexington, MA 02420 USA

Phone: (781) 860-0501, (781) 862-8422

Fax: (781) 860-0599

Internet: www.fshsociety.org

e-mail: daniel.perez@fshsociety.org