Evicenter
P&T meetings

RP-A501 (AAV9.LAMP2B)

Danon disease

Regulatory submission
BLA planned for late 2028
Last updated
300 sources

Product information and disease descriptionPlace of product in therapyApproaches to treatmentCurrent treatment options and standard of care

Multidisciplinary supportive care

11 evidence topics · 7 sources

Consensus statement: multidisciplinary care at referral centers

“A multidisciplinary care approach, including a cardiology, neurology, genetics, ophthalmology, rehabilitation medicine and physical therapy, should be used to alleviate the multisystem effects of the disease.” (opens the source at this quote in a new tab)

“It is recommended that patients with Danon disease be assessed and receive follow-up care at referral centers where a multidisciplinary team can provide a comprehensive management strategy.” (opens the source at this quote in a new tab)

“Ideally, integrated multidisciplinary assessment and follow-up care should be organized for these challenging patients, along the model suggested for Fabry disease.” (opens the source at this quote in a new tab)

Physical therapy for skeletal myopathy

“Physical therapy and educational support are also recommended for skeletal myopathy and cognitive impairment, respectively.” (opens the source at this quote in a new tab)

“Neuromuscular examination to assess for signs of skeletal myopathy at initial evaluation and during follow-up visits is recommended for all patients.” (opens the source at this quote in a new tab)

“Physicians should aim to prevent progressive loss of muscle strength and flexibility in affected patients through standard physical therapy and light exercise.” (opens the source at this quote in a new tab)

“Assessment of muscle strength, particularly the proximal muscles of the shoulder, neck, and legs, should be performed during scheduled physical examination visits.” (opens the source at this quote in a new tab)

Rehabilitation strategy suggested by muscle activation testing

“Such central activation deficits suggest that rehabilitation strategies that optimize muscle activation, in conjunction with volitional muscle strengthening exercises, may be justified.” (opens the source at this quote in a new tab)

Developmental assessment and educational support

“Comprehensive developmental assessment with a view to initiating early educational support and interventions should be performed in all male individuals with Danon disease and in female individuals as clinically indicated.” (opens the source at this quote in a new tab)

“Intellectual difficulties should be anticipated and identified for early intervention.” (opens the source at this quote in a new tab)

“We suggest that comprehensive neuropsychological exams may be useful to assess other neurocognitive problems such as attention-deficit hyperactivity and autism-spectrum disorders.” (opens the source at this quote in a new tab)

“Enrollment in a rehabilitation center for educational, psychological, and social support is suggested.” (opens the source at this quote in a new tab)

Psychiatric assessment

“At the time of psychiatric evaluation, 9 participants (69.2%) met criteria for at least one psychiatric disorder” (opens the source at this quote in a new tab)

“Of importance, it underscores the need to refer Danon patients to psychiatric assessment.” (opens the source at this quote in a new tab)

Neuropsychological evaluation across the life span: single case

“This case study further explores the nature of the deficits related to Danon disease and highlights the benefits of neuropsychological evaluation to guide functional interventions and maximize level of independence across the life span.” (opens the source at this quote in a new tab)

Ophthalmic surveillance and low vision support

“Ophthalmologic assessment for evidence of pigmentary retinopathy is recommended at initial evaluation and during follow-up care in all patients.” (opens the source at this quote in a new tab)

“Use of low vision aids” (opens the source at this quote in a new tab)

“Low vision aids such as magnifiers and closed circuit television may provide useful reading vision for individuals with reduced central acuity and constricted visual fields.” (opens the source at this quote in a new tab)

“Impact on vision and rate of progression of retinal disease is not well understood.” (opens the source at this quote in a new tab)

Ophthalmic findings in a systematic review of 70 patients

“Retinal abnormalities were the most common finding (81%), typically midperipheral and peripheral salt-and-pepper pigmentary changes.” (opens the source at this quote in a new tab)

Genetic counseling and family planning

“Genetic counseling should be offered to all affected families to ensure they are aware of the inheritance pattern, reproductive risks, and technologies available to those who wish to pursue further testing before, during, and after pregnancy.” (opens the source at this quote in a new tab)

“First, cascade genetic screening of relatives should be recommended at the time of diagnosis; second, family planning strategies should be introduced when a patient reaches reproductive age.” (opens the source at this quote in a new tab)

Social work and care coordination

“Ensure appropriate social work involvement to connect families w/local resources, respite, & support.” (opens the source at this quote in a new tab)

“Coordinate care to manage multiple subspecialty appointments, equipment, medications, & supplies.” (opens the source at this quote in a new tab)

Physical activity guidance

“Exercise testing with the use of treadmill ergometry or cardiopulmonary exercise testing is useful for risk stratification and to provide guidance about physical activity.” (opens the source at this quote in a new tab)

“No specific guidelines exist for individuals with non-sarcomeric cardiomyopathy.” (opens the source at this quote in a new tab)