AGENDA

Friday, February 27th

7:30am

Registration Open & Continental Breakfast

Registration Desk C

Breakfast in Grand Ballroom D

8:30am

Welcome and Conference Overview

Terry Thompson, KDA President

Grand Ballroom C

8:45am

Kennedy's Disease: Symptoms and Health Effects

Christopher Grunseich, MD

Grand Ballroom C

9:15am

Respiratory Issues with Kennedy's Disease

Noah Lechtzin, MD

Grand Ballroom C

10:00am

Break

Grand Ballroom C Foyer

10:30am

Managing Falls: How to Optimize your Functioning to Prevent falls

Louie Lee, MD, MCSP & Joe Shrader, PT

Grand Ballroom C

11:30am

SBMA Genetics: International Survey on Genetic Literacy and Awareness in Patients

Shinichiro Yamada, MD, PhD

Grand Ballroom C

11:45am

NIH's All of Us Project: Prevalence of the KD Genetic Mutation

Dan Tsuma

Grand Ballroom C

12:00pm

Kennedy's Disease Patient and Family Forum

Lunch Sponsored by: AnnJi Pharmaceutical

Grand Ballroom C

1:30pm

The Engine that Drives the Research

Lew Myers

Grand Ballroom C

1:50pm

Raising Awareness and Access to Local Care

Kim Slowe & Luca Zampedri

Grand Ballroom C

2:30pm

KD Tips and Tricks: At Home and on the Road

Jameson Parker & Dale Traxler

Grand Ballroom C

3:00pm

Break

Grand Ballroom C Foyer

3:30pm

Breakout Sessions

KD Men- Salon 11

Partners/Caregivers- Salon 12

Carriers- Salon 13

5:00pm

KDA Grant & Awardees Reception

Upper Pool Deck

Dinner on Your Own

Saturday, February 28th

7:45am

Registration Open & Continental Breakfast

8:30am

Conference Updates and Plan for the Day

Terry Thompson, KDA President

Grand Ballroom C

8:40am

Introducing the Canadian Protocol for Managing Kennedy's Disease

Kerry Schellenberg, MD

Grand Ballroom C

8:50am

Managing KD Symptoms: Advice from Clinicians on Managing Fatigue and Other Topics, Followed by Q&A

Chris Grunseich MD, Kerri Schellenberg, MD, Tahseen Mozaffar, MD, Carlo Rinaldi, MD, Angela Kokkins, BSN, RN, Luca Zampedri, Noah Lechtzin, MD, Abdullah AlQahtani, MD, MPH

Grand Ballroom C

9:50am

Break

Grand Ballroom C Foyer

10:20am

The Latest in Mobility Devices: Talk & Demo

Jim Kissling

Grand Ballroom C

10:45am

Beyond Exoskeletons: HAL-Based Cyborg Neurorehabilitation for SBMA – Clinical and Observational Evidence on Combined Therapy with Leuprorelin

Takashi Nakajima, MD, PhD

Grand Ballroom C

11:15am

KD-101: Introduction to KD Biology

Ed Meyertholen, PhD

Grand Ballroom C

11:45am

Lunch and Learn

Sunday, March 1st

7:30am

Registration Open & Continental Breakfast

Session 1: AR Function and Dysfunction

8:30am

When AR Goes Nuclear - and Wrong

Maria Pennuto, PhD

8:55am

TBD

Xavier Salvatella

9:20am

Muscle-Specific Inhibition of AR Co-Activators: Results of a Pre-Clinical Study

Manuela Basso, PhD

9:30am

Optional Breakout Sessions

Newly Diagnosed- Salon 11

KD Men- Ballroom D

Partners/Caregivers- Salon 12

Carriers- Salon 13

9:55am

Androgens and neuronal activity synergistically act on Androgen Receptor to modulate the immediate transcriptional response to stimulation

Emanuela Zuccaro

10:10am

Break

10:35am

TBD

Al La Spada, MD, PhD, FACMGG

Session 2: Mechanisms of Tissue Specificity

11:00am

Mechanisms of Neuromuscular Degeneration

Andrew Lieberman, MD, PhD

11:25am

When Muscles Change Their Identity: NMJ Failure and Fiber-Type Remodeling in SBMA

Helen Miranda

11:50am

Researchers Lunch- Sponsored by Terry and Kathy Thompson

1:00pm

Poster Session 1

Even numbers

2:30pm

A Journey from Transcription to Therapeutic Target

Carlo Rinaldi, MD

2:55pm

TBD

Janghoo Lim

3:20pm

Modeling spinal and bulbar muscular atrophy using patient-derived neuromuscular organoids

Sara De Vincentiis

3:35pm

Break

Session 3: Proterostesis

3:55pm

Drosophila models of Kennedy’s Disease

Sokol Todi

4:20pm

Mechanisms of nuclear AR aggregation in SBMA

Diane Merry, PhD

4:45pm

Discovery and Development of Androgen Receptor N-Terminus Domain-Binding Molecular Glue Degraders for the Treatment of Kennedy's Disease

Marjana Khalil

5:00pm

Clinical features and genetic analysis from the Indigenous founder population for SBMA in western Canada

Gerald Pfeffer

5:30pm-Wrap up & End the Day

Monday, March 2nd

7:45am

Registration Open & Continental Breakfast

Session 4: Emerging Therapeutic Targets

8:30am

Development of Small Molecule PRMT6 Inhibitors for the Treatment of Spinal Bulbar Muscular Atrophy

Rachel Harding

8:55am

From Pathogenic Mechanisms to Emerging Therapeutic Strategies: Targeting Mutant Androgen Receptor Toxicity in Spinal and Bulbar Muscular Atrophy (SBMA)

Angelo Poletti

9:20am

CD38 activity and inflammation in SBMA

Heather Montie

9:45am

Novel siRNA therapeutics for polyglutamine disease

Masahisa Katsuno, MD, PhD

10:05am

Break

10:40am

Reduction of mutant AR in SBMA by small molecule mRNA splicing modulators

Mary McMahon

10:55am

Investigating RNA editing dysregulation in SBMA and other polyglutamine disorders

Alfina Ambra Speciale

11:10am

A Muscle-Targeted Gene Therapy for Kennedy’s Disease

Annalucia Darbey

11:25am

Evaluation of the 3rd generation brain penetrant HSP90 inhibitor ATS-033 as a drug candidate for SBMA

Nathan Bechouche

11:40am

Muscle Mitochondrial Dysfunctions and Systemic Lipidomic Remodeling in SBMA

Mariarita Galbiati

11:55am

Lunch

1:00pm

Poster Session 2

Odd Numbers

Session 5: Clinical Trials Readiness

2:30pm

Exploring the Prevalence and Clinical Spectrum of Androgen Receptor Repeat Expansions in a Population-Based Cohort

Dan Tsuma

2:45pm

Biomarker development in spinal and bulbar muscular atrophy

Christopher Grunseich, MD

3:10pm

Break

3:25pm

A Phase 3 trial evaluating the efficacy and safety of AJ201 (Rosolutamide) in ambulatory participants with spinal and bulbar muscular atrophy: ROMA trial

Andy Chen

3:40pm

TBD

Vissia Viglietta, MD, PhD

4:05pm

ARV-027 induces peripheral PolyQ-AR degradation and positively impacts muscle strength and endurance in a murine model of SBMA

James Gregory, PhD

4:30pm- Wrap up & End the Day