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

Research and Markets
3-min read
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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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