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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 weekly 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)
DUVYZAT® (givinostat) On MARCH 21, 2024, the FDA approved an eighth treatment specifically for Duchenne muscular dystrophy. This is a nonsteroidal treatment indicated for DMD in people 6 years of age and older, regardless of their genetic variant.
ELEVIDYS® (delandistrogene moxeparvovec-rokl) ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated in individuals at least 4 years of age:
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)
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