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Angelman Syndrome (AS) Market Assessment 2024: US And EU5 Epidemiology, Treatment Landscape, Unmet Needs, Emerging Therapies, And Value & Access
(MENAFN- GlobeNewsWire - Nasdaq) 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:
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
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