Bardet-Biedl Syndrome – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030

Bardet-Biedl Syndrome (BBS) is a rare autosomal recessive defect, belonging to the family of ciliopathies. Children born from consanguineous marriages are mostly affected. The primary manifestations of BBS are rod-cone dystrophy (90 to 93%), postaxial polydactyly (62-82%), obesity (73-91%), learning disabilities (62%), genital abnormalities (59-98%), and renal abnormalities both morphological and functional (53%). The visual prognosis for children with BBS is poor with a mean age of legal blindness of 15 years. Significant weight gain begins within the first year and becomes a lifelong issue for most individuals. A majority of individuals have significant learning difficulties, but only a minority have severe impairment on IQ testing. Renal disease is a major cause of morbidity and mortality. Clinical findings establish the diagnosis of BBS. Multiple genes are known to be associated with BBS: BBS1, BBS2, ARL6 (BBS3), BBS4, BBS5, MKKS (BBS6), BBS7, TTC8 (BBS8), BBS9, BBS10, TRIM32 (BBS11), BBS12, MKS1 (BBS13), CEP290 (BBS14), WDPCP (BBS15) SDCCAG8 (BBS16) LZTFL1 (BBS17), and BBTP1 (BBS18). BBS is typically inherited autosomal recessive, but up to 15% of patients do not have identifiable mutations in known genes. Both interfamilial and intrafamilial phenotypic variability exists. Carrier testing and prenatal testing are possible if the disease-causing mutations in a family are known.

Incidence rates vary from country to country like in North America is found to be 1 to 2 cases per 150,000 and in Europe, it is to be 1 to 1.5 cases per 150,000 live births.

 

The competitive landscape of Bardet-Biedl 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 Bardet-Biedl 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.

Bardet-Biedl 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: Bardet-Biedl 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

 

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