Stargardt Disease Overview

Stargardt Disease Overview

Stargardt disease is an inherited disorder that leads to progressive vision impairment, usually starting in childhood or teenage years. It is considered complex when the severity of retinal damage or related complications makes treatment more difficult or increases the likelihood of serious outcomes. The condition primarily targets the macula, the central part of the retina responsible for detailed vision, causing symptoms like blurred sight, trouble reading, difficulty recognizing faces, and, at times, color vision loss. As the condition advances, central vision continues to deteriorate, greatly affecting daily life.

There is currently no cure for Stargardt disease. Treatment efforts aim to manage symptoms and slow disease progression through tools such as visual aids, UV-blocking eyewear, and occupational training to support everyday tasks. Ongoing research is exploring therapies such as gene editing, retinal implants, and other advanced interventions. Early diagnosis and genetic counseling are vital for helping patients and families understand the condition and plan ahead, especially since the disease follows an autosomal recessive inheritance pattern. Though vision loss cannot be reversed, consistent medical care and adaptation strategies can help individuals maintain a good quality of life.

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Stargardt Disease Epidemiological Segmentation (2020–2034, 7MM)

• Diagnosed prevalent cases
  
  

• Regional case distribution
  
  

• Age of onset segmentation
  
  

• Classification by type
  
  

• Symptom-based segmentation
  
  

• Total diagnosed case count
  
  

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Stargardt Disease Epidemiology

In 2023, the total diagnosed prevalence of Stargardt disease across the 7 major markets (7MM) was estimated at approximately 53,000 cases.

• The United States represented the largest share with 43% of the cases.
  
  

• The EU4 (France, Germany, Italy, Spain) and the UK accounted for 41%, and Japan made up the remaining 16%.
  
  

• In the U.S., individuals under 20 years of age had the highest number of new diagnoses by age group.
  
  

Stargardt Disease Market Insights

• Market Size (2023, 7MM): Around USD 27 million
  
  

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Market Drivers

• Progress in gene and stem cell-based therapies is opening new treatment avenues.
  
  

• Improved understanding of genetic mutations behind the disease is advancing research.
  
  

• Targeting the retinal pigment epithelium (RPE) has created novel therapeutic strategies.
  
  

• Increased focus on innovative technologies is accelerating drug development.
  
  

Market Challenges

• Misdiagnosis and underdiagnosis, especially in adults with late-onset symptoms
  
  

• Disease variability, which complicates diagnosis and treatment plans
  
  

• High cost of cutting-edge therapies, limiting access for many patients
  
  

• Lack of awareness in some regions, leading to delayed diagnosis and care
  
  

Emerging Therapies for Stargardt Disease

• Emixustat
  
  

• Tinlarebant
  
  

• MCO-010
  
  

• IZERVAY (Zimura)
  
  

• And others
  
  

Key Companies in the Stargardt Disease Market

• Kubota
  
  

• Belite Bio
  
  

• Nanoscope Therapeutics
  
  

• Astellas
  
  

• Among others
  
  

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