Santhera Seeks Participants for DMD Study, Makes Available EAP and Online DMD Resource

With a focus on respiratory care in Duchenne muscular dystrophy (DMD), Santhera Pharmaceuticals has made available new resources for individuals with the disease. In addition, the company is seeking individuals to participate in a phase 3 clinical trial to test a respiratory drug in people with DMD.

SIDEROS trial seeks participants

Researchers are looking for boys and men age 10 years and older with DMD to participate in a phase 3 clinical trial.

The study, called SIDEROS, is designed to help researchers determine the safety and efficacy of idebenone (brand name Raxone). Idebenone is an experimental drug in development by Santhera Pharmaceuticals that researchers hope will delay the loss of respiratory function in DMD.

Idebenone is an oral tablet. Scientists hypothesize it may work by improving the way muscles utilize fuel to power movement.

SIDEROS is designed to assess whether idebenone can slow decline in respiratory function in boys and men with DMD. It will compare the efficacy of idebenone to placebo in individuals who currently are receiving treatment with steroids (prednisone or deflazacort).

In order to be eligible to participate, individuals:

  • may be ambulatory (able to walk) or non-ambulatory
  • must have been taking a stable dose of corticosteroids for at least 12 months
  • must not be participating in another trial, have taken any investigational drug within 90 days of screening or have taken certain approved drugs
  • must not need daytime ventilator assistance
  • must not have any other non-DMD respiratory illness — such as asthma, COPD, bronchitis, emphysema or pneumonia — at the time of screening
  • must meet additional study criteria

Study participants will be randomly assigned to groups that will receive either idebenone or placebo. After completing the study, all participants will have the opportunity to enroll in an open-label extension study in which everyone will receive treatment with idebenone.

The study is expected to last 22 months, during which time participants will be required to make nine clinic visits at three-month intervals. These visits will include a series of lung function tests to determine changes in lung capacity. Throughout the study, participants will continue to see their regular doctors for routine care.

Dozens of trial sites are open across the United States. Support is available for travel and hotel accommodations.

Visit for more information about travel and accommodations support.

To learn more or to inquire about participation, email, visit Santhera’s website at or visit and enter NCT02814019 in the search box.

Expanded access program: BreatheDMD

In February, Santhera Pharmaceuticals launched an expanded access program in the United States for boys and men with DMD to receive idebenone outside of a clinical trial.

Through the program, called BreatheDMD, eligible patients who are 10 years or older and experiencing decline in respiratory function can obtain access to idebenone at no cost.

If you think you or your child may benefit from enrollment in BreatheDMD, speak to your physician. For details about BreatheDMD, visit

Take a Breath DMD

For helpful information on respiratory health, Santhera Pharmaceuticals has also launched a new online resource called Take a Breath DMD.

At the Take a Breath DMD website, you can read about how DMD affects breathing, learn how respiratory muscle function is measured and get tips for day-to-day life with DMD. You can also sign up to receive news alerts, clinical updates and emails.

Santhera is a proud national sponsor of MDA.

To learn more, visit