DMD APPROVED TREATMENTS & STUDIES

On  MARCH 21, 2024, the FDA approved an eighth treatment for Duchenne  - Italfarmaco’s givinostat (DUVYZAT®). WE WILL POST AN OFFICIAL LISTING HERE SOON!

AGAMREE® (vamorolone) oral suspension 40 mg/mL is a structurally unique corticosteroid indicated for the treatment of Duchenne muscular dystrophy in patients 2 years of age and older. If you or someone you know is interested in AGAMREE, visit www.AGAMREE.com or reach out to Catalyst Pathways at 1-833-422-8259.

AMONDYS45® (casimersen) - Exon 45 skipping treatment granted accelerated approval in the United States. This treatment is for patients who have a mutation amenable to skipping exon 45. It is a once eekly injection. Clinical trials currently underway globally (more info at https://www.sarepta.com/products-pipeline/clinical-trials.) 

SareptAssist 1-888-SAREPTA (1-888-727-3782)

ELEVIDYS® (delandistrogene moxeparvovec-rokl)  ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated in individuals at least 4 years of age:

• For the treatment of Duchenne muscular dystrophy (DMD) in patients who are ambulatory and have a confirmed mutation in the DMD gene. 
• For the treatment of DMD in patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin.  Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). 

EMFLAZA® (deflazacort) - Corticosteroid approved in the United States for DMD patients age 2 years and older. If you or someone you know is interested in Emflaza, please speak with your physician about obtaining a prescription or contact PTC Cares (https://ptccares.com/; 1-844-478-2227)

EXONDYS 51® (eteplirsen) - Exon 51 skipping treatment granted accelerated approval in the United States. This treatment is for patients who have a mutation amenable to skipping exon 51. It is a once weekly injection. Clinical trials are currently underway globally (more info at https://www.sarepta.com/products-pipeline/clinical-trials.) 

SareptAssist 1-888-SAREPTA (1-888-727-3782)

VILTEPSO® (viltolarsen) - Exon 53 skipping treatment approved in Japan in March 2020 and given accelerated approval in the United States in August 2020.  Phase 3 clinical trials continue in the USA, Canada, and globally.  European Commission (EC) granted Orphan Drug Designation in June 2020. This treatment is for patients who have a mutation amenable to skipping exon 53. It is a once weekly injection. Patient Support Hub - NS Support 1-833-NSSUPRT (833-677-8778) Monday - Friday 8 a.m. - 8 p.m. 

VYONDYS 53® (golodirsen) - Exon 53 skipping treatment granted accelerated approval in the United States. This treatment is for patients who have a mutation amenable to skipping exon 53. It is a once weekly injection. Clinical trials are currently underway globally (more info at https://www.sarepta.com/products-pipeline/clinical-trials.)

SareptAssist 1-888-SAREPTA (1-888-727-3782)