This course is available as a stand-alone purchase, part of the Pediatric Neuromuscular Course Bundle, or the 2024 Annual Meeting Video Collection.

Explore the effect of myostatin inhibitors on patients with SMA; examine the state of various antisense oligonucleotide therapies; and survey other small molecule therapies in early studies as well as “add-on” intrathecal gene therapy for older patients with SMA.

OBJECTIVES:
1) Describe how myostatin inhibitors affect SMA patients; 2) compare different antisense oligonucleotide treatments for SMA; 3) identify new small molecule therapies being tested for SMA; 4) explain how intrathecal gene therapy might help older SMA patients as an additional treatment

ACCREDITATION STATEMENT
The AANEM is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

CREDIT DESIGNATION :
The AANEM designates this enduring material for a maximum of 1.5 AMA PRA Category 1 CreditsTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

All relevant financial relationships have been mitigated.

Speakers:

Meeta Cardon, MD 

• Life Sciences Consulting- consultant

Ashley N. Stanley-Copeland, MD

• Novartis, Catalyst- consulting fee

Jenna Klotz, MD

• No disclosures