Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM

Limb-girdle Muscular Dystrophy

Becker Muscular Dystrophy

Prednisone

Northwestern University

Weekly Steroids in Muscular Dystrophy

Open Label Safety and Efficacy of Once Weekly Steroid in Patients With LGMD and Becker Muscular Dystrophy

mg/dL, 0-unlimited, higher score indicates worse outcome

% , 0-100, higher score indicates worse outcome

cholesterol levels - mg/dL, higher levels indicate worse outcomes

units/L, 0-unlimited, higher scores indicate worse outcome

Force Vital Capacity (% of predicted value), decrease in FVC indicates declining respiratory function.

Northstar Assessment for Dysferlinopathy
- score out of 58, range from 0 to 58, higher score indicates greater functional ability.

number of meters walked in 6 minute period. Higher values indicate more motor function.

time in seconds to walk/run 10 meters , less time to run indicates greater motor function

Performance of Modified Upper Limb Module - Yes or No questions, ability to raise 50 gram, 100 gram, 200 gram, 500 gm weights above the head and to the mouth.

upper extremity assessment, scoring between 1- 6, lower score indicates more upper extremity function

Lower extremity assessment, score from 1-10, lower score indicates more function.

Motor Muscle Testing: score of 1 - 5 , higher score indicates more muscle strength.

MRI of leg muscles to measure changes in muscle

whole dexa body scan to assess bone density with Z scores (more negative z score indicates increased risk for fractures)

whole body dexa scans to assess lean mass % (0- 100 %). Increase lean mass % is the desired outcome.

Senda Ajroud-Driss

Associate Professor of Neurology (Neuromuscular Disease)

Givinostat oral suspension (10 mg/mL) twice daily in a fed state

Placebo oral suspension (10 mg/mL) twice daily in a fed state

Duchenne Muscular Dystrophy

givinostat

suspension manufactured to mimic givinostat

placebo

Neuromuscular Research Center UC Davis Department of Physical Medicine and Rehabilitation

David Geffen School of Medicine - UCLA Neurology

Rady Children's Hospital center - UCSD Department of Neuroscience

Children's Hospital Colorado

Connecticut Children's Medical Center - Division Neurology

Child Health Research Institute - Department of Pediatrics

Nemours Children's Hospital

MD Rare Disease Research, LLC

University of Iowa Children's Hospital

Wayne State University - University Pediatrics - Children's Hospital of Michigan

University of Minnesota - Department of Neurology

Washington University School of Medicine in St Louis - Department of Neurology

Shriners Hospitals for Children

The Children's Hospital of Philadelphia Colket Translational Research Building

Virginia Commonwealth University Childrens Hospital of Richmond at Virginia Commonwealth University

University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology

Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN)

Kinsmen Research Centre - Alberta Children's Hospital - Alberta Health Services

The University of British Columbia, Children's and Womens Health Centre of BC Branch

Holland Bloorview Kids Rehabilitation Hospital

CHU de Nantes - Hotel-Dieu - Hopital Nord Laennec, rez-de-chausse haut ail Ouest

Hopital Armand Trousseau I-Motion, Plateforme d'essais cliniques pédiatriques

Universitatsklinikum Essen - Kinder und Jugendmedizin Neuropadiatrie

Klinik un Policlinik fur Kinder und Jugendmedizin - Universitatsklinikum Hamburg Eppendorf

Klinikum der Uniersitat Munchen - Campus Innenstadt

Institute of Neurology - Schneider Children's Medical Center of Israel Kaplan, 14

IRCCS Istituto G.Gaslini, U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative

A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS di Neurologia Pediatrica

Fondazione IRCCS Istituto Neurologico Carlo Besta

Centro Clinico NeMO Fondazione Serena ONLUS Area SUD

Ospedale Pediatrico Bambin Gesù, Malattie Neuromuscolari e Neurodegenerative

Fondazione Policlinico Universitario "A.Gemelli", UOC Neuropsichiatria Infantile

Leiden University Medical Center LUMC

Radboud University Medical Centre

Clinic of Neurology and Psychiatry for Children and Youth - Neurology Department Dr. Subotic 6a,

Hospital Sant Joan de Déu - Neuromuscular Pathology Unit

Hospital Materno-Infantil - Passeig de la Vall d'Hebron

Hospital Universitario Virgen del Rocio

Hospital Universitari i Politécnic de la Fe - Servicio Neurologia

Alder Hey Children's Hospital NHS Trust

UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD

The John Walton Muscular Dystrophy Research Centre - Freeman Hospital - Newcastle University - Institute of Genetic Medicine

The Robert Jones and Agnes Hunt Orthopaedic Hospital - NHS Foundation Trust

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

the primary endpoint is the mean change in 4 standard stairs climb test results before and after 18 months of treatment of givinostat versus placebo

the mean change in time to rise from floor

the mean change in fat fraction of vastus lateralis muscles at MRS

the mean change in North Star Ambulatory Assessment

the mean change of muscle strength evaluated by knee extension, elbow flexion as measured by hand-held myometry (HHM)

muscular dystrophies/triglyceride

Weekly Steroids in Muscular Dystrophy

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

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