DUCHENNE MUSCULAR DYSTROPHY (DMD) TREATMENT
About the Disease
Duchenne muscular dystrophy (DMD) is a genetic, progressive form of muscular dystrophy that occurs primarily in males. It is caused by genetic changes in the DMD gene and is inherited in an X-linked recessive pattern. X-linked means the gene for the condition is located on the X-chromosome. In males, one altered copy of the gene is enough to cause DMD. DMD causes progressive weakness and muscle atrophy. In early signs of the disease, DMD may cause a delayed ability to sit, stand, or walk, and can make learning to speak difficult for the patient.
About our Patient Care
Our dedicated Care Team will work with the physician's office and insurance on a patient's behalf so that our patients have more time to focus on their treatment and quality of life. We are here to listen, provide counseling on treatment, including side-effect management, and assist with sourcing third-party financial assistance.
In addition, Orsini is the only specialty pharmacy with access to all medications for Duchenne Muscular Dystrophy.
As part of treatment, we provide:
Dedicated Care Team
We provide prescribers and patients with a dedicated care team for each of our disease state programs. This includes a primary pharmacy contact, clinical education, side-effects management, scheduled refill calls, and a direct phone, fax and email address.
In-Home Patient- and Family-Centric Care
We administer a nationwide nursing network, allowing patients to pursue in-home treatment or receive training on self-administrated drugs. Our nurses are ideally positioned to establish a partnership with patients and their families in order to provide clinical education and coaching that strengthen the patient's capacity to achieve therapy goals.
Shipping and Coordination of Care
We provide delivery of medications and necessary medical supplies and manage the coordination of care between prescribers, sites of care, nurses and patients to ensure dispenses and drug delivery align with the date and location of service.
Financial Assistance
We successfully identify and assist enrolling eligible patients into any and all manufacturer co-pay and foundation support programs in order to minimize a potential financial burden to patients.
24/7/365 Access to a Pharmacist
We provide prescribers and patients access to a pharmacist 24/7/365 to ensure patients are always cared for.
Supported Treatments
Click on the specialty drug name below to expand and see more information.
AMONDYS 45 (casimersen)
ELEVIDYS (delandistrogene moxeparvovec-rokl)
Brand Name | ELEVIDYS (delandistrogene moxeparvovec-rokl)
Manufacturer | Sarepta Therapeutics, Inc.
Route of Administration | Suspension for intravenous (IV) infusion
Approved Indication | Treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene and is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.
Self-Administration | No
Product Website | www.sarepta.com
EMFLAZA (deflazacort)
Brand Name | EMFLAZA®
Manufacturer | PTC Therapeutics, Inc.
Route of Administration | Oral
Approved Indication | Treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. See full prescribing information.
Self-Administration | Yes
Product Website | www.emflaza.com
EXONDYS 51 (eteplirsen)
Brand Name | EXONDYS 51
Manufacturer | Sarepta Therapeutics, Inc.
Route of Administration | Intravenous (IV) infusion
Approved Indication | Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping
Self-Administration | No
VYONDYS 53 (golodirsen)
Brand Name | VYONDYS 53
Manufacturer | Sarepta Therapeutics, Inc.
Route of Administration | Intravenous (IV) infusion
Approved Indication | Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping
Self-Administration | No
VILTEPSO (viltolarsen) Injection
Brand Name | VILTEPSO
Manufacturer | NS Pharma
Route of Administration |Intravenous (IV) infusion
Approved Indication | Treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping
Self-Administration | No
Product Website | www.viltepso.com
AMONDYS 45, ELEVIDYS, EXONDYS 51, and VYONDYS 53 are registered trademarks of Sarepta Therapeutics, Inc.
EMFLAZA is a registered trademark of PTC Therapeutics Inc.
VILTEPSO is a registered trademark of NS Pharma, Inc.