Page 2 - 2021 Spring CMTA Report - Special Research Edition

CELEBRATING STAR RESEARCH

ACCELERATING
CONTENTS
RESEARCH

EMPOWERING

PATIENTS

A MESSAGE FROM THE CEO ................................................................................................... 3
BUILDING AND PAYING FOR A STAR ................................................................................ 4-5
CROSS-TYPE INITIATIVES ......................................................................................................6-8
CMTA-FUNDED STUDY FOR CMT1B ...................................................................................... 8
INHERITED NEUROPATHY CONSORTIUM EXPLAINED ..................................................9
CMTA | STAR TIMELINE OF GROWTH ..............................................................................10-11
STAR’S PORTFOLIO OF DEMYELINATING PROJECTS ..............................................12-13
STAR BIOTECH ALLIANCE PARTNERS FOR CMT1A ...................................................14-15
DEMYELINATING PROJECTS PROGRESS CHART ......................................................16-17
NEW MOUSE MODELS OF CMT1X ........................................................................................17
STAR 1A TIMELINE ................................................................................................................. 18-19
PHASE 3 CLINICAL TRIALS FOR 1A ...............................................................................20-21
CLINICAL TRIALS — THE ROAD TO DRUG APPROVAL ...............................................22
THE CHALLENGE OF RARE DISEASES ...............................................................................23
WHY WE GIVE ..............................................................................................................................24
THE CMTA I STAR FUNDING PROCESS ..............................................................................25
STAR’S PORTFOLIO OF AXONAL PROJECTS ..........................................................26-27
AXONAL PROJECTS PROGRESS CHART ...................................................................28-29
THE CMTA’S TYPE 2 GENE DISCOVERY INITIATIVES ....................................................30
THE CMTA’S TYPE 2 GENE REPLACEMENT THERAPY INITIATIVES ..........................31
CMT2A RESEARCHERS ON THE WAY TO BEING TRIAL-READY ..............................32
WONDERING HOW YOU CAN GET INVOLVED? ..............................................................33
ACCELERATING RESEARCH. EMPOWERING PATIENTS ...............................................34

THE CMTA REPORT

Marcia Semmes, Executive Editor
Karlyn Rosen Aires, Designer
Dana Schwertfeger, Contributing Editor
Teresa Carroll, Contributing Writer
Kenneth Raymond, Contributing Writer

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