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Disease management

Current approaches

Now there is a treatment option available for Alexander disease, marking an important advancement that reflects a growing understanding of the disease and expands management beyond symptomatic care.17 At the same time, a multidisciplinary approach focused on alleviating symptoms and enhancing quality of life remains essential to disease management.1 Because of the rare nature of Alexander disease, there are a limited number of medical experts who specialize in confirming the diagnosis and developing a disease management plan, which means that affected people may need to travel long distances to consult with specialists.65

Additional approaches to disease management include therapies or devices to compensate for some of the physical (eg, seizures, dysphagia, mobility), speech (eg, dysphonia, dysarthria), and cognitive (eg, developmental delay, behavioral problems) difficulties experienced by people living with Alexander disease.1,38 

A black and white photo of a boy and a man on a beach leaning on a rock together with the boy’s shirt in red.
Images depicted are not actual patients.

An FDA-approved prescription medicine is available for pediatric and adult patients with Alexander disease.17

 

Learn more about a treatment for Alexander disease

Icon of a pill bottle and blister pack to represent neurological medications that manage seizures and other symptoms.

NEUROLOGICAL MEDICATIONS

To manage seizures, muscle spasticity, dystonia, pain, and other neurological symptoms1,38

Icon of a banana, apple, and nutrition guide for addressing feeding difficulties.

NUTRITIONAL SUPPORT AND DIETARY MODIFICATIONS

To address feeding difficulties due to dysphagia and/or recurrent vomiting21,38

Icon of a stomach with a gastric tube.

Gastronomy tube

To ensure adequate nutrition, minimize aspiration risk, and administer medications for those with severe dysphagia38

Icon of lungs to represent respiratory interventions.

RESPIRATORY Interventions

To address respiratory insufficiency, including infection prevention, airway maintenance, and mechanical support38

Icon of a tablet and a pill to represent GI and urinary medications.

GI/URINARY Medications/interventions

To control reflux, vomiting, drooling, constipation, and urinary retention/incontinence1,38

Icon of a smartphone with a plus sign in a caption to represent augmentative and alternative communication strategies.

Augmentative and alternative communication strategies

To assist with communication impairments21

Icon of two overlapping heads to represent occupational therapy.

Occupational Therapy

To assist those with physical, cognitive, and/or developmental impairments to maintain their ability to perform everyday tasks21

Icon of an arm pulling a pulley with a weight on it for physical therapy.

Physical THERAPY

To improve range of motion and strength, preserve motor skills, and maximize mobility21,38

Icon of a walker that helps people with mobility issues.

Assistive Devices

To improve mobility, such as orthotics, braces, gait trainers, wheelchairs, walkers, lifts, and standers38

Icon of a person in bed with a crescent moon overhead to represent sleep hygiene optimization.

Sleep Hygiene Optimization

To address sleep disorders and appropriate interventions for sleep apnea38

Icon of a head with a gear in it and another behind it to represent neurophysical support.

Neuropsychological support

To address learning and cognitive development, and to develop a plan to optimize learning strategies21

Icon of two bones and their joint to represent orthopedic exams.

Orthopedic exams and interventions

To evaluate and treat scoliosis, hip dysplasia and dislocation, and low bone mass/fractures38

MULTIDISCIPLINARY TEAM

According to the 2015 GLIA consensus guidelines, the use of a collaborative multidisciplinary team of healthcare providers to address the range of clinical symptoms has the potential to improve care in people with leukodystrophies.66 This team may include1,38:

  • Primary care physicians
  • Palliative care specialists
  • Neurologists
  • Neurosurgeons
  • Feeding specialists
  • Gastroenterologists
  • Physical and occupational therapists
  • Geneticists
  • Urologists
  • Radiologists
  • Pulmonologists
  • Orthopedic surgeons
  • Speech-language pathologists
  • Social workers

The transition from pediatric to adult care may be especially challenging in individuals with rare disorders; the establishment of standard protocols for the management of transition may help to facilitate a seamless integration from child-centered to adult-centered care.67