Angelman Syndrome (AS) Market Assessment 2024: US and EU5 Epidemiology, Treatment Landscape, Unmet Needs, Emerging Therapies, and Value & Access


Dublin, Sept. 27, 2024 (GLOBE NEWSWIRE) -- The "Angelman Syndrome Market Assessment: Epidemiology, Treatment Landscape, Unmet Needs, Emerging Therapies, and Value & Access" report has been added to ResearchAndMarkets.com's offering.

Angelman Syndrome market landscape report combines primary (KOL interviews) and secondary market research to empower strategic decision-making and provide a complete view of the market. Itincludes a disease overview, epidemiology (US and EU5), current treatment, unmet needs, pipeline and access and reimbursement chapter.

Topics covered in this report:

  • Disease overview: Review the disease pathophysiology and potential druggable targets
  • Epidemiology: Understand prevalence, diagnosed and drug-treated prevalence of the population and key market segments
  • Current treatment: Understand the treatment decision tree and strengths and weaknesses of current on-label and off-label treatment
  • Unmet needs: Identify opportunities to address treatment or disease management gaps
  • Pipeline analysis: Compare current and emerging therapy clinical development strategy; their performance on efficacy, safety, and delivery metrics; and their potential to address unmet needs
  • Value and access: Review the evidence needed to assess and communicate value to key stakeholders (e.g., providers, payers, regulators) and learn what competitors have done or are doing

Key companies mentioned:

  • Roche
  • Ionis
  • Biogen
  • Ultragenyx
  • Neuren Pharmaceuticals
  • Foundation for Angelman Syndrome Therapeutics (FAST)
  • Encoded Therapeutics
  • Transformatx
  • Ovid Therapeutics

Key drugs mentioned:

  • Melatonin
  • Trazodone
  • Clonazepam
  • Lamotrigine
  • Levetiracetam
  • Topiramate
  • Clonidine
  • Atomoxetine (Strattera)
  • Risperidone
  • Aripiprazole (Abilify)
  • Polyethylene glycol
  • Omeprazole
  • Rugonersen
  • ION582
  • GTX-102
  • NNZ-2591
  • Alogabat
  • GTP-220
  • Gaboxadol

Key Topics Covered:

1. DISEASE OVERVIEW

  • A rare, neurogenetic disorder characterized by developmental delays, seizures, and a happy and excitable demeanor
  • AS molecular subtypes
  • The role of UBE3A in the disease mechanism of AS
  • The role of UBE3A in the hypothesized pathogenesis of AS

2. EPIDEMIOLOGY & PATIENT POPULATIONS

  • Disease definition
  • G6 diagnosed prevalent cases of Angelman Syndrome by region
  • Diagnosed incident and prevalent populations of AS in the U.S. and EU5
  • AS epidemiology and subtypes
  • Proportion of AS patients with each molecular subtype

3. DIAGNOSIS & CURRENT TREATMENT

  • Diagnosis overview
  • Diagnostic pathway for AS patients
  • AS requires lifelong medication courses and therapy
  • Treatment goals for AS - physician versus caregiver priorities
  • Proportion of AS patients with difficult-to-treat seizures
  • AS treatment involves a multidisciplinary care team collaborating with caregivers
  • AS care team roles and responsibilities
  • AS key characteristics throughout life course
  • Physician insights on current AS treatment approaches
  • Key treatment dynamics that will shape disease management and novel therapy use in AS
  • Must-know AS market dynamics
  • Large opportunity for a therapy that will address neurodevelopmental outcomes
  • Important dynamics of AS market evolution

4. UNMET NEED

  • Overview
  • Top unmet needs in AS
  • Physician perspectives on unmet needs in AS

5. PIPELINE ANALYSIS

  • Overview
  • Comparison of ongoing AS disease-modifying therapy clinical trials
  • Comparison of ongoing AS non-disease-modifying therapy clinical trials
  • Preclinical AS gene therapy and delivery platforms
  • Novel "HItap" AAV delivery platform
  • Preclinical GTP-220 summary
  • Preclinical AS gene therapy pipeline
  • Preclinical research on AS gene therapies
  • Physician insights on gene therapy options
  • Failed AS trials and important drivers of future AS product differentiation
  • Ovid Therapeutics OV101 Phase 3 efficacy data
  • Important drivers of future AS product differentiation
  • Attributes that will drive uptake for emerging AS therapies

6. VALUE & ACCESS

  • Comparison of gene therapy pricing
  • Comparison of ASO therapy pricings
  • Key reimbursement and access considerations for emerging gene therapies in AS
  • Key insights from the gene therapy market
  • Innovative payer coverage gene therapy insights
  • Financial hurdles in AS diagnosis and treatment
  • Mean annual financial impact on caregivers for AS
  • Mean annual healthcare costs per patient with development epileptic encephalopathies

7. METHODOLOGY

For more information about this report visit https://www.researchandmarkets.com/r/cow56e

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