An unintended or unwanted outcome from use of a medication.

A disease caused by a mutation in the dystrophin gene that results in progressive muscle weakness. BMD is similar to Duchenne muscular dystrophy (DMD), but is less severe.

A disease that affects the heart muscles’ ability to function properly.

An organization that established the largest long-term observational study to track the progression of DMD in people over time, including their use of medical services and any issues they experienced. The study took place globally and enrolled over 550 participants.

The molecule that carries the genetic information for an organism’s development and functioning.

A muscle disorder caused by a mutation in the dystrophin gene (“DMD gene”) that leads to a progressive decline of muscle function, as well as heart problems and lung issues.

The ability of a treatment to provide long-term benefits.

Part of a group of proteins (a protein complex) that all work together to strengthen and protect muscles as they contract and relax.

A region of DNA with genetic information for making a protein.

Dystrophin that’s produced by exon-skipping therapies. This form of dystrophin is larger than the micro-dystrophin that’s produced by gene therapies. With exon-skipping therapy, the muscle cells of a person with Duchenne make a blueprint for their dystrophin protein that’s 84–97% (for the most common mutations) as long as the full-length dystrophin made by someone who doesn't have DMD.

A therapy that helps people with Duchenne "skip over" exons so that compatible exons can connect and create a shorter but usable dystrophin protein.

Changes in a person’s DNA sequence.

A therapy that inserts an engineered micro-dystrophin gene into a person with Duchenne to help their muscle cells produce a micro-dystrophin protein. With gene therapy, the dystrophin blueprint made in muscle cells of a person with Duchenne is only 32–40% as long as the full-length dystrophin made by someone without DMD.

Sequences of DNA containing information for making proteins that perform specific biological functions.

Analyzing a person’s DNA sample in a laboratory to detect changes or mutations that may be linked to genetic disease.

A specific pattern of movements used by people with Duchenne to raise themselves from the floor, using their hands as support to push themselves to an upright position.

A mutation that occurs when adding or removing genetic information from a gene, resulting in a semi-functional protein. In-frame mutations can result in BMD.

A micro-dystrophin protein created through gene therapy that’s 32–40% as long as the dystrophin protein made by someone without DMD.

Standardized tests typically given to a person with Duchenne to assess the impact of their DMD based on their performance in specific activities requiring motor skills and strength. Motor function tests have been validated in clinical trials to monitor DMD progression. They can also be used to monitor progress of DMD treatment and support ongoing reimbursement with payors.

The goal of OT is to help people with Duchenne maximize their functional independence in everyday activities. OT can also help with addressing tasks for school or work, maintaining skills for hobbies and leisure, and facilitating social integration with peers.

A mutation that occurs when adding or removing genetic information from a gene, resulting in a non-functional (dysfunctional) protein.

The goal of PT is to help people with Duchenne preserve their range of motion, motor function and overall strength. People with Duchenne should receive PT to encourage their mobility and help prevent or reduce risk of muscle contractures. PT for people with Duchenne typically involves passive stretching exercises at the ankles, knees, and hips, four to six times a week. These can help prevent or reduce contractures of the Achilles tendons, iliotibial bands, and hip flexors. In later DMD stages, additional stretching may be needed at the wrist, hands, and neck.

Using multiple drugs for one or more conditions in a single person.

Watching a person for any adverse reactions after a treatment infusion.

Biological molecules that perform specific functions for the body.

Medicines that help reduce inflammation in the body. The steroids used to treat DMD are systemic, which means that they circulate through the bloodstream to various sites in the body. Steroids are often used for people with Duchenne to help slow their muscle loss and reduce severity of spine deformities (scoliosis).

A motor function test that measures how long it takes for a person with Duchenne to climb four stairs.

A motor function test that measures how long it takes for a person with Duchenne to run or walk 10 meters (about 32 feet).

A motor function test that measures how long it takes for a person with Duchenne to go from lying on their back to standing.

The time it takes for a medication to be fully eliminated from a person’s body, often used in clinical trials to ensure accurate results.

A laboratory technique used to identify proteins and help predict severity of a disease.

A genetic disease associated with mutations in genes on the X chromosome.

A motor function test that measures how far a person with Duchenne can walk in six minutes on a flat surface.