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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