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

 

SLC13A5 deficiency is a rare autosomal recessive disorder caused by mutations in the sodium-coupled citrate transporter gene SLC13A5. It is characterized by infantile seizures and neurodevelopmental delays. Patients with SLC13A5 citrate transporter disorder are initially identified by the multiple types of seizures 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 including problems standing or walking independently, as well as abnormalities in tooth enamel. Problems with tone are also reported with chronic low tone but also 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 as well as the time course of developmental milestones.

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

The competitive landscape of SLC13A5 Deficiency includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are being 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

Comments

Post a Comment

Popular posts from this blog

Acne Vulgaris – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Image
  Acne vulgaris, a prevalent pilosebaceous unit inflammatory skin disorder, follows a persistent course. Typically presenting as papules, pustules, or nodules, it primarily occurs on the face but can also impact the upper arms, trunk, and back. The development of acne results from the sebaceous glands' heightened sensitivity to normal androgen levels, aggravated by Cutibacterium acnes (formerly known as Propionibacterium acnes), a bacterial species that triggers inflammation. The pathogenesis of acne vulgaris involves a complex interplay of factors such as the activation of sebaceous glands by androgens, imbalances in the pilosebaceous follicle microbiome, and cellular immune responses. Genetic predisposition and dietary influences also contribute to the condition's onset and progression. The initial stage is the micro-comedo, a small plug primarily composed of corneocytes in the lower follicular infundibulum. Microcomedones evolve into various acne lesions, including closed co...
Read more

Metabolic Syndrome – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Image
  Metabolic syndrome refers to a clustering of multiple disorders that collectively increase an individual's susceptibility to atherosclerotic cardiovascular disease, insulin resistance, diabetes mellitus, as well as vascular and neurological complications such as cerebrovascular accidents. The underlying causes of metabolic syndrome typically include excess weight, obesity, a sedentary lifestyle, and genetic predisposition. This syndrome exerts detrimental effects on various body systems. Insulin resistance, a key component of metabolic syndrome, leads to microvascular damage, increasing the risk of endothelial dysfunction, vascular resistance, hypertension, and inflammation of vessel walls. Bariatric surgery is recognized as the most effective standalone therapy for metabolic syndrome. However, patients undergoing this procedure require long-term follow-up to mitigate potential surgical, nutritional, and psychiatric complications. Thelansis’s “Metabolic Syndrome Market Outl...
Read more

Type 1 Diabetes Mellitus (T1D) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Image
  Type 1 Diabetes Mellitus (T1D) is characterized as an autoimmune disorder resulting in the autoimmune destruction of insulin-producing pancreatic beta cells. This condition necessitates life-long insulin replacement, administered through multiple daily insulin injections, insulin pump therapy, or an automated insulin delivery system. The absence of insulin can lead to the development of Diabetic Ketoacidosis (DKA), a life-threatening condition. The most common symptoms associated with this disease are polyuria and polydipsia. Effective management of T1D involves: Multiple daily insulin injections Insulin pump therapy An automated insulin delivery system in conjunction with glucose monitoring Continuous Glucose Monitoring (CGM) is the preferred method. However, all individuals with T1D should be capable of performing Blood Glucose Monitoring (BGM) via capillary blood if CGM is not available. Acute complications of diabetes primarily include hypo...
Read more