Duchenne Muscular Dystrophy Questions and Answers

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Idebenone is an investigational medicine (formulated as tablets) for the treatment of Duchenne muscular dystrophy (DMD).

Based on current data, idebenone is believed to work by acting on mitochondria - structures in cells involved in energy production.

Idebenone is undergoing clinical trials to evaluate its safety and efficacy in DMD and is not approved by the FDA.

In clinical trials to date, the most common side effects seen with idebenone were: Nasopharyngitis (sore throat and nasal passages), diarrhea and headache. You should discuss the side effects with your treating physician to see if taking idebenone in the BreatheDMD program would be suitable for you.

Mitochondria are tiny structures found within muscles and are thought to provide energy needed for muscles to contract, including those involved with breathing.

An expanded access program is a route through which eligible patients can gain access to a medicine under investigation before it is approved by regulatory authorities and there is no suitable alternative treatment for the medical condition. A clinical trial is conducted under controlled conditions and its primary purpose is to answer a clinical question about an investigational medicine and to gain approval to make the product widely available in the US.

When patient enrollment in a clinical trial is not possible (for example when there are no ongoing clinical trials or when a patient is not eligible for a trial), patients may be able to receive the investigational medicine, when appropriate, through expanded access.

Yes, you first need to contact your treating physician about BreatheDMD. They will be able to answer any initial questions regarding your participation and support your subsequent assessment by a local BreatheDMD center.

You may be eligible for BreatheDMD if you are in a clinical trial for other medications for DMD. If you’re already in a clinical trial involving idebenone or are eligible for another trial with idebenone, you will not be able to participate in BreatheDMD. You should talk with your treating physician to check your eligibility.

Yes – your treating physician will refer you to a participating center to evaluate if you are eligible to participate in the BreatheDMD expanded access program. They will be able to review the eligibility criteria for participation and contact the local BreatheDMD center to process your enrollment.

Forced vital capacity, or FVC, is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. FVC is used to help determine both the presence and severity of lung diseases.

Peak Expiratory Flow, or PEF, is the maximum flow of air when a person breathes out as forcibly and hard as they can. It is used as a measure of lung function.

No – Although BreatheDMD has been set-up to help patients gain access to the investigational medicine idebenone, not everyone who wishes to participate in the BreatheDMD program will be accepted. This could be due to a variety of reasons, for example: not meeting all the eligibility criteria. You should speak with your treating physician regarding participation in BreatheDMD.

You and your parent/guardian will be contacted by the participating center, to confirm you meet the eligibility requirements. You and your parent/guardian will then be asked to attend an Enrollment Visit. During this visit, there will be specific medical examinations performed to confirm your eligibility. You will also need to sign a consent form to confirm your acceptance of participation and also for allowing data collection during the program.

After this, you will return to the participating center approximately every 6 months, where they will perform routine testing.

Eligible patients may receive idebenone until treatment is discontinued for clinical reasons or until the drug is approved by the FDA and insurance reimbursement is available, whichever comes first. Treatment may also be discontinued if the sponsor determines to terminate the program.

Idebenone, the investigational medicine used in BreatheDMD, will be provided free-of-charge. However, expenses associated with transportation to/from your BreatheDMD physician’s office, as well as any other expenses incurred during your participation in this program are the responsibility of the patient and family.

Data collected that is personally identifiable during your participation in BreatheDMD program will only be accessible to medical staff directly involved in the program. Permission will be sought for the sharing of any data from patient and/or parent guardian. All data collected will be anonymized to protect your identity and is subject to current US data protection legislation.

You should contact your treating physician about participating in BreatheDMD.

Expanded Access Program (EAP)

Idebenone is a medicine that is under investigation for the treatment of DMD. It has not been approved by the US Food and Drug Administration (FDA) and the safety and efficacy continue to be evaluated in clinical trials.

Participating Physicians

To find your closest BreatheDMD center, roll your mouse over the map below. If your treating physician is not listed here, you may ask them to refer you to your nearest BreatheDMD center.

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