Adult Considerations in Spinal Muscular Atrophy: Building Clinician and Patient Medical Teams


Claudia A. Chiriboga, MD, MPH, FAAN
Professor of Neurology and Pediatrics
Professor of Neurology and Pediatrics
Division of Pediatric Neurology, Columbia University Medical Center, New York, NY
Claudia A. Chiriboga, MD, MPH, FAAN
Professor of Neurology and Pediatrics

Dr. Chiriboga is Professor of Neurology and Pediatrics at Columbia University Irving Medical Center (CUIMC) and former Interim Director of the Division of Child Neurology, Departments of Neurology and Pediatrics, Vagelos College of Physicians and Surgeons. She is also former Chief of Pediatric Neurology at Columbia University’s affiliate hospital, Harlem Hospital Center.

Dr. Chiriboga was born in Peru and educated in Argentina, where she graduated with Honors from the Faculty of Medicine at the National University of Buenos Aires in 1982. Dr. Chiriboga completed training in pediatrics at St. Luke’s-Roosevelt Hospital in 1985. After completing her training in child neurology in 1988 at the Neurological Institute and Babies’ Hospital of Columbia-Presbyterian Medical Center, Dr. Chiriboga joined the Division of Child Neurology at NewYork-Presbyterian/Columbia University Irving Medical Center. Subsequently, she completed a Neuroepidemiology fellowship at the Gertrude H. Sergievsky Center and received a master’s in public health from the Mailman School of Public Health, Columbia University. Dr. Chiriboga serves/has served on committees of the NIH, CNS, and the American Board of Psychiatry and Neurology (ABPN). She is a diplomate of the ABPN in Child Neurology.

Dr. Chiriboga’s current research focuses on Spinal Muscular Atrophy (SMA) clinical trials. Past areas of research, including NIH funding, have focused on congenital infections and neurodevelopmental disorders, including cerebral palsy and effects of perinatal exposures. Her clinical practice focuses on neuromuscular disorders as well as developmental, neurobehavioral neurology, and migraine, and on the treatment of spasticity.

Vanessa Battista, DNP, MBA, MS, RN, CPNP-PC
Senior Nursing Director, Palliative Care, Dana Farber Cancer Institute
Department of Psychosocial Oncology & Palliative Care, Pediatric Nurse Practitioner, Boston, MA
Vanessa Battista, DNP, MBA, MS, RN, CPNP-PC

Vanessa Battista, DNP, MBA, MS, RN, CPNP-PC, CHPPN, FPCN is a board-certified Pediatric Nurse Practitioner (PNP) and currently serves as Senior Nurse Director of Palliative Care at the Dana Farber Cancer Institute. Previously, she practiced as a PNP on the Pediatric Advanced Care Team (PACT) at the Children’s Hospital of Philadelphia (CHOP). She also served at the neuromuscular centers at CHOP, the Columbia University Medical Center, and the Children’s Hospital of Boston. Dr. Battista was clinical faculty at the Boston College Connell School of Nursing where she developed the Pediatric Palliative Care Masters subspecialty program.

Dr. Battista also serves as a board member and consultant for various organizations that provide services for patients and families living with life-threatening diseases. She lectures and teaches at the local, national, and international level, and is a member of the End-of-Life Nursing Education Consortium (ELNEC) pediatric faculty. Dr. Battista is an active reviewer for publications and grants, and has authored several book chapters and articles on various aspects of pediatric palliative care and neuromuscular disease.

Dr. Battista was a Jonas Nursing Scholar, and the recipient of the ELNEC Award for Excellence, the Distinguished Young Alumni Award from Columbia University, and the DNP Leadership Award from Johns Hopkins University. She also earned Clinical Master status among advanced practice providers at CHOP. She earned her bachelor’s degree from Boston College in psychology, with minors in health sciences and faith, peace, and justice studies; her nursing degree and master’s degree from Columbia University School of Nursing; and completed a certificate in Pastoral Ministry at the Boston College School of Theology and Ministry. Dr. Battista obtained her Master of Business Administration and Doctor of Nursing Practice from Johns Hopkins University.

Tina Duong, PT, PhD
Physical Therapist
Director, Clinical Outcomes Research & Development, Division of Neuromuscular Medicine, Stanford University, Stanford, CA
Tina Duong, PT, PhD

Dr. Duong is the director of clinical outcomes research and development at Stanford University Neuromuscular division. Clinically, she is a physical therapist with 20 years of clinical experience in evaluating, treating, and validating clinical outcomes for patients with neuromuscular disease. She is involved in ongoing studies and developments on initiatives in treatments for neuromuscular disease, novel trial designs, and outcomes development. Prior to her arrival at Stanford University in 2015, she directed clinical evaluations and training for an international network, Cooperative International Neuromuscular Research Group (CINRG), in Washington, DC.

Her clinical and research interests in developing, refining, and understanding outcome measures in neuromuscular disease contributes to her determination in improving efficacy endpoints for clinical trials as well as interpreting these endpoints on the changing phenotype in neuromuscular diseases. Her area of research focuses on the impact of exercise and physical activity on disease physiology and quality of life. She hopes to integrate technology and digital biomarkers as a complementary tool to performance-based measures used in trials, in the clinic, and remotely.

She enjoys working on multi-disciplinary teams, mentorship, and creating patient-focused solutions to improve the quality of life for all neuromuscular patients. Her interests and hobbies include hiking, running, gardening, and working with technology and design products to make the world more accessible for individuals with different abilities.

Statement of Need

Adults now make up more than a quarter of patients with spinal muscular atrophy (SMA), a debilitating genetic disease characterized by progressive muscle weakness and atrophy, but advances in research, supportive care, and disease-modifying therapies have largely centered on pediatric populations. Consequently, adults with SMA often face “fragmented” care pathways that are particularly problematic when transitioning from pediatric to adult care.

In this educational webcast, expert faculty will discuss treatment needs of adult patients with SMA and the unique challenges and experiences of those patients transitioning from pediatric to adult care. The webcast will also include insights about how to navigate systems and

Learning Objectives

  • Evaluate the burden of SMA on adult patients including clinical, quality of life (QoL), and psychological/psychosocial outcomes.
  • Develop best practice interdisciplinary strategies to meet the needs of patients with SMA transitioning from pediatric to adult care.
  • Integrate appropriate therapeutic approaches to care for adult patients with SMA.

Financial Support

Supported by an educational grant from Genentech, a member of the Roche Group.

Target Audience

Neurologists, neuromuscular specialists, physician associates (PAs), nurse practitioners (NPs), physical therapists, and adult patients with SMA

Credit Information

Jointly Accredited Provider

In support of improving patient care, CME Outfitters, LLC, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

Interprofessional (IPCE) 1.0

This activity was planned by and for the healthcare team, and learners will receive 1.0 Interprofessional Continuing Education (IPCE) Credit for learning and change.

Physicians (ACCME) 1.0

CME Outfitters, LLC, designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nurses (ANCC) 1.0

This activity is designated for 1.0 contact hour.

Note for California Nurses

This continuing nursing education activity was approved by the California Board of Registered Nursing. CME Outfitters, LLC’s provider number is CEP15510.

Pharmacists (ACPE) 1.0

This application-based activity is approved for 1.0 contact hour (0.10 CEUs) of continuing pharmacy credit.
Activity UAN: JA0007185-0000-22-159-H01-P

PAs (AAPA) 1.0

CME Outfitters, LLC, has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with AAPA CME Criteria. This activity is designated for 1.0 AAPA Category 1 CME credits. Approval is valid until the expiration date listed above. PAs should only claim credit commensurate with the extent of their participation.


Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 medical knowledge MOC point in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.

MIPS Improvement Activity

Completion of this accredited CME activity meets the expectations of an Accredited Safety or Quality Improvement Program (IA_PSPA_28) for the Merit-based Incentive Payment Program (MIPS). Clinicians should submit their improvement activities by attestation via the CMS Quality Payment Program website.

Royal College MOC

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Disclosure Declaration

It is the policy of CME Outfitters, LLC, to ensure independence, balance, objectivity, and scientific rigor and integrity in all of their CE activities. Faculty must disclose to the participants any relationships with commercial companies whose products or devices may be mentioned in faculty presentations, or with the commercial supporter of this CE activity. CME Outfitters, LLC, has evaluated, identified, and mitigated any potential conflicts of interest through a rigorous content validation procedure, use of evidence-based data/research, and a multidisciplinary peer review process.

Dr. Chiriboga reports the following financial relationships:

Advisory Board/Consultant: Genentech, Inc.; Novartis Pharmaceuticals Corporation; PTC Therapeutics; and Roche

Research Support: AveXis, Inc./Novartis Gene Therapies; Biogen; and Roche

Speakers Bureau: Biogen Colombia; Genentech, Inc.; and Roche

Dr. Battista reports the following financial relationships:

Advisory Board: Biogen and Novartis Pharmaceuticals Corporation

Speakers Bureau: Novartis Pharmaceuticals Corporation

Dr. Duong reports the following financial relationships:

Advisory Board: ActiGraph, LLC; Astellas Pharma Inc.; Avidity Biosciences; Biogen; Dyne Therapeutics; Genentech, Inc.; Pfizer Inc.; Roche; Sanofi; Sarepta Therapeutics, Inc.; and Scholar Rock, Inc.

Consultant: Astellas Pharma Inc.; ATOM; Genentech, Inc.; Roche; and TRiNDS

Grants: Biogen and Ionis Pharmaceuticals, Inc.

Research Support: Biogen

Speakers Bureau: Genentech, Inc. and Sarepta Therapeutics, Inc.

Disclosures were obtained from the following peer reviewer and CME Outfitters, LLC, staff, with no disclosures to report:

  • Michael Franks, APRN, AGACNP-BC, FNP-BCD (peer reviewer)
  • Thomas Mitchell (planning committee)
  • Kellie Busby, PharmD (planning committee)
  • Susan H. Yarbrough, CHCP (planning committee)
  • Sandra Caballero, PharmD (planning committee)
  • Sharon Tordoff (planning committee)

Faculty of this CE activity may include discussions of products or devices that are not currently labeled for use by the FDA. The faculty have been informed of their responsibility to disclose to the audience if they will be discussing off-label or investigational uses (any uses not approved by the FDA) of products or devices.

Obtaining Credit

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Adult Considerations in Spinal Muscular Atrophy: Building Clinician and Patient Medical Teams
Event Date: 11/09/2022