SLC13A5 Deficiency – Market outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2020 To 2030


 SLC13A5 deficiency is a rare autosomal recessive disorder caused by mutations in the sodium-coupled citrate transporter gene SLC13A5. Infantile seizures and neurodevelopmental delays characterize it. Patients with SLC13A5 citrate transporter disorder are initially identified by the multiple types of attacks that begin within the first week of life. This rare disease is due to changes (mutations) in the SLC13A5 gene (solute carrier family 13, member 5). Seizures begin within a few days of birth, which are often refractory to medications and most patients remain on anti-seizure medications throughout life. Additional symptoms include limited ability to speak, slow motor development, problems standing or walking independently, and abnormalities in tooth enamel. Problems with tone are also reported with chronic low style and periodic episodes of body stiffening and post stiffening weakness. Siblings with the same genetic mutation show differences in the severity of symptoms. Variations include the type and frequency of seizures and the time course of developmental milestones.

 

Based on the literature review, ~220 patients are diagnosed in the USA, and the disease affects both males and females equally.

 

The competitive landscape of SLC13A5 Deficiency 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 SLC13A5 Deficiency 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.

SLC13A5 Deficiency 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: SLC13A5 Deficiency – Market outlook, Epidemiology, Market Forecast, and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          TSHA-105         Taysha Gene Therapies, Inc.     Phase 1

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