Alström Syndrome – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030

 Alström Syndrome is an ultra-rare multisystem genetic disorder caused by pathogenic variants of the ALMS1 gene. The disease is relentlessly progressive in nature which can result in premature death. Misdiagnosis is common. Because so many of the features of Alström Syndrome develop as children get older, more than seventy percent of the known Alström Syndrome patients have received previous misdiagnoses, and almost half of these have received multiple misdiagnoses. It progresses to severe visual impairment by the end of the second decade in 75%. Obesity develops in early childhood. Progressive bilateral sensorineural hearing loss is more variable, often presenting with high-frequency loss in the first decade, but not detectable until much later in a minority. More than 60% of individuals with AS develop cardiomyopathy. This can present as potentially reversible dilated cardiomyopathy in infancy, or presenting de novo or recurrence in adolescence with progression to a restrictive pattern. Insulin resistance is present from infancy- and progression to glucose intolerance is partly related to the degree of obesity. Molecular genetic testing of ALMS1, the only gene in which mutations are known to cause Alström syndrome, is estimated to detect mutations in 70%-80% of individuals of northern European descent, and approximately 40% worldwide.

Prevalence range from 1:10,000 in communities where first cousin marriages are customary to fewer than 1:1,000,000 in populations with low levels of consanguineous marriages.

The competitive landscape of Alström Syndrome includes country-specific approved and pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are tracked and supplemented with analyst commentary.

KOLs insights of Alström Syndrome across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

Alström Syndrome Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard) which Data Inputs with sourcing, Market Event, and Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Read more: Alström Syndrome – Market outlook, Epidemiology, Market Forecast, and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          Setmelanotide   Rhythm Pharmaceuticals, Inc.    Phase 3

2          PBI-4050          Liminal BioSciences Ltd.            Phase 2