Lamzede (velmanase)
Research Synopsis
- - Lamzede (velmanase) is an enzyme replacement therapy approved in Europe for the treatment of alpha-mannosidosis, a rare genetic disorder characterized by a deficiency of the enzyme alpha-mannosidase, affecting approximately 1 in 500,000 to 1 in 1,000,000 births.! - Recent studies indicate that velmanase alfa is effective in treating non-neurological symptoms in patients with mild to moderate forms of the disease, with a high responder rate of 87% observed in one study after 12 months of treatment.! - Long-term efficacy data show significant biochemical and functional improvements in patients treated with velmanase alfa over up to four years, leading to improved health outcomes such as reduced serum oligosaccharide levels and better performance on functional tests like the stair climb test.! - Early treatment initiation, particularly during childhood, has been linked to better functional outcomes, reinforcing the importance of timely diagnosis and intervention in managing alpha-mannosidosis.! - Ongoing research, including large-scale registries like SPARKLE, aims to collect long-term data on the safety and effectiveness of velmanase alfa under routine clinical settings, providing insights into disease progression and treatment benefits.! - Some studies have examined the impact of genetic variations on the development of anti-drug antibodies in response to velmanase therapy, finding that while some patients develop these antibodies, treatment efficacy remains largely unaffected.! - Innovative monitoring techniques, such as high-performance liquid chromatography (HPLC) and nuclear magnetic resonance (NMR), have been validated for tracking oligosaccharide levels in patients undergoing treatment, enhancing the understanding of therapy response.! - Overall, the therapeutic profile of velmanase alfa demonstrates both safety and efficacy for managing alpha-mannosidosis, with most adverse events reported as mild to moderate, suggesting it is generally well tolerated by patients.! - Patient and caregiver experiences highlight the emotional and mental burdens associated with alpha-mannosidosis, emphasizing the need for improved healthcare access and awareness for timely diagnosis and treatment.! - Future research is essential to confirm long-term outcomes and establish velmanase alfa as a standard therapy for alpha-mannosidosis, particularly given the complex nature of the disorder and variations in individual patient responses.!
Lamzede (velmanase)
Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis.
London, UK
2 hours ago
1 Received
- Alpha-mannosidosis is an extremely rare genetic disorder (about 1 in 1,000,000 births) caused by a lack of the enzyme alpha-mannosidase, leading to various clinical symptoms due to the buildup of specific sugar molecules.
- The enzyme replacement therapy (ERT) called velmanase alfa is approved in Europe for treating non-neurological symptoms in patients with mild to moderate forms of the disease.
- A new responder analysis revealed that after 12 months of treatment, 87% of patients on velmanase alfa showed positive responses across multiple health domains, indicating significant effectiveness and the need for early and ongoing treatment.
$100 - $150
Hourly Rate
Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis.
London, UK
2 hours ago
1 Received
- Long-term outcome data indicate that velmanase alfa is effective for treating alpha-mannosidosis (AM), showing significant improvements in both biochemical and functional measures over up to 4 years of treatment.
- Analysis involved 33 patients (14 adults, 19 children) who continued treatment after clinical trials, with key findings including substantial reductions in serum oligosaccharide levels and improved performance in a stair climb test.
- Results suggest that early treatment during childhood leads to better functional outcomes, and no serious adverse events caused treatment discontinuation, highlighting the therapy's safety and effectiveness.
$100 - $150
Hourly Rate
Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial.
London, UK
2 hours ago
1 Received
- A phase III, double-blind, randomized trial evaluated the efficacy and safety of velmanase alfa (VA) in patients with alpha-mannosidosis (AM) over 52 weeks, with a follow-up for those who switched from placebo.
- Twenty-five patients were assigned to receive either VA or a placebo, with key measures being changes in serum oligosaccharide levels and performance on a stair-climb test.
- Results showed a significant reduction in serum oligosaccharides and improvement in stair-climb performance for those on VA, especially if treatment started early in childhood, highlighting VA's potential as an effective therapy for AM.
$100 - $150
Hourly Rate
Hearing impairment as an early sign of alpha-mannosidosis in children with a mild phenotype: Report of seven new cases.
London, UK
2 hours ago
1 Received
- * Diagnosis typically involves measuring enzyme activity and detecting mannose-rich oligosaccharides, with genetic confirmation through MAN2B1 mutations; enzyme replacement therapy has been available since 2018.
- * A study of seven individuals with syndromic hearing loss and learning disabilities highlighted the challenges of diagnosing AM, underscoring the potential of exome sequencing to aid early identification in subtle cases, improving treatment and management.
$100 - $150
Hourly Rate
Disease progression of alpha-mannosidosis and impact on patients and carers - A UK natural history survey.
London, UK
2 hours ago
1 Received
- Alpha-mannosidosis is a rare genetic disorder caused by a deficiency in alpha-mannosidase, leading to varied symptoms and progression among patients.
- A survey of nine patients in the UK revealed differences in mobility and overall health-related quality of life (HRQoL), with those less mobile reporting lower HRQoL, while treatments like hematopoietic stem cell transplantation and enzyme replacement therapy improved their well-being.
- The results highlight the emotional and mental strain on caregivers, emphasizing the need for early diagnosis and treatment to improve outcomes for both patients and their families.
$100 - $150
Hourly Rate
Use of the Bruininks-Oseretsky test of motor proficiency (BOT-2) to assess efficacy of velmanase alfa as enzyme therapy for alpha-mannosidosis.
London, UK
2 hours ago
1 Received
- Alpha-mannosidosis is a rare genetic disorder affecting balance, immunity, and cognition, with new enzyme replacement therapy (velmanase alfa) being introduced to treat symptoms.
- A study on this therapy showed that children experienced a significant improvement in motor skills (13% increase) over time, while adults showed little to no change.
- Overall, children treated with velmanase alfa demonstrated continued skill development, potentially leading to greater independence and better quality of life.
$100 - $150
Hourly Rate
Early biochemical effects of velmanase alfa in a 7-month-old infant with alpha-mannosidosis.
London, UK
2 hours ago
1 Received
- Alpha mannosidosis is a rare genetic disorder caused by the deficiency of the enzyme alpha mannosidase, which leads to the buildup of certain sugars in the body.
- Until recently, the main treatment option was hematopoietic stem cell transplantation, but in 2018, a new enzyme replacement therapy called velmanase alfa was approved for treating mild to moderate symptoms.
- A study on a 7-month-old patient who received velmanase alfa showed significant reductions in the levels of harmful sugars in urine and serum, suggesting the enzyme is effective in managing the disease's symptoms.
$100 - $150
Hourly Rate
The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis.
London, UK
2 hours ago
1 Received
- - SPARKLE is a registry for alpha-mannosidosis aimed at gathering long-term safety and effectiveness data on velmanase alfa, the first enzyme replacement therapy for the disorder, particularly for non-neurologic symptoms in patients with mild to moderate cases.
- - The registry is a multicenter, multinational study, enrolling patients since 2020, and will track outcomes for up to 15 years, focusing on safety and effectiveness under routine clinical care.
- - Primary safety measures include monitoring adverse events and antibody development, while effectiveness is assessed through various health outcomes, providing insights into the disease's progression and natural history in treated and untreated patients.
$100 - $150
Hourly Rate
Caregivers' and Physicians' Perspectives on Alpha-Mannosidosis: A Report from Italy.
London, UK
2 hours ago
1 Received
- Alpha-mannosidosis is a rare genetic disorder that primarily affects children, often leading to delays in diagnosis due to its complex symptoms and the need for multiple specialist referrals.
- The study highlights the experiences of two mothers from different backgrounds, one in a city and the other in a rural area, showing how access to healthcare and timely diagnosis can vastly differ and impact the care journey for their children.
- The report emphasizes the need for increased awareness among healthcare providers and improved support systems for both patients and their caregivers, especially in rural settings where access to specialized care may be limited.
$100 - $150
Hourly Rate
The Application of HPLC-FLD and NMR in the Monitoring of Therapy Efficacy in Alpha-Mannosidosis.
London, UK
2 hours ago
1 Received
- Alpha-mannosidosis is a rare genetic disorder resulting from a deficiency in the enzyme α-D-mannosidase, leading to the accumulation of undigested sugars in cells, which are excreted in urine.
- The study involved analyzing urinary oligosaccharides from a patient undergoing a new enzyme replacement therapy using advanced techniques like HPLC and NMR, finding a significant decrease in these sugars after treatment.
- Results indicated the therapy's effectiveness, with a two-fold decrease in oligosaccharides at one month and a ten-fold decrease at four months, supporting both HPLC-FLD and NMR as reliable methods for monitoring treatment outcomes in alpha-mannosidosis.
$100 - $150
Hourly Rate
Relationship between genotype/subcellular localization subgroups, antidrug antibody detection, and long-term velmanase alfa treatment outcomes in patients with alpha-mannosidosis.
London, UK
2 hours ago
1 Received
- - Alpha-mannosidosis (AM) is a genetic disorder causing lysosomal enzyme deficiency, leading to the buildup of specific sugars; Velmanase alfa (VA) is the first enzyme replacement therapy targeting AM's non-neurological symptoms.
- - A study involving 33 patients on VA explored if genetic subgroups (G1, G2, G3) influenced antibody response (antidrug antibodies or ADAs) and related infusion reactions, finding that G1 and G2 were more likely to develop ADAs but most patients experienced mild or no infusion-related reactions.
- - Despite some patients developing ADAs, the overall treatment effects on serum oligosaccharides and clinical outcomes were similar across ADA-positive and ADA-negative
$100 - $150
Hourly Rate
Velmanase Alfa-tycv.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Long-term safety and efficacy of velmanase alfa treatment in children under 6 years of age with alpha-mannosidosis: A phase 2, open label, multicenter study.
London, UK
2 hours ago
1 Received
- - Alpha-mannosidosis (AM) is a rare genetic disorder caused by the deficiency of an enzyme, leading to the buildup of certain sugars, with symptoms that can be severe and often go undiagnosed until late childhood.
- - Velmanase alfa (VA) is the first enzyme replacement therapy approved in Europe for treating non-neurological symptoms of AM, with research suggesting earlier treatment could lead to better outcomes.
- - A phase 2 study involving five children under 6 years old found that long-term VA treatment was generally safe, well-tolerated, and resulted in improvements in health indicators, although the small sample size limits broader conclusions.
$100 - $150
Hourly Rate
First experience of combined enzyme replacement therapy and hematopoietic stem cell transplantation in alpha-mannosidosis.
London, UK
2 hours ago
1 Received
- The case study discusses the first instance of bridge therapy for an infant with alpha-mannosidosis, who started enzyme replacement therapy (ERT) at 5 months old before undergoing hematopoietic stem cell transplantation (HSCT).
- Eight ERT infusions were given prior to HSCT and continued for 90 days post-transplant, showing promising results in slowing disease progression and reducing harmful substances in the urine and plasma.
- The report emphasizes that early diagnosis and timely treatment are crucial for effectively managing alpha-mannosidosis and improving patient outcomes.
$100 - $150
Hourly Rate
Exploring the feasibility of using the ICER Evidence Rating Matrix for Comparative Clinical Effectiveness in assessing treatment benefit and certainty in the clinical evidence on orphan therapies for paediatric indications.
London, UK
2 hours ago
1 Received
- The study assesses the challenges in evaluating clinical evidence for orphan therapies, especially in pediatric patients, due to limited available data and the complexities of clinical trials.
- The researchers used the ICER Evidence Rating Matrix to evaluate the clinical benefit and certainty of eleven orphan therapies designated by the EMA, focusing on those approved between January 2017 and March 2020.
- Results showed that two therapies (dinutuximab beta and nusinersen) had a "substantial" net health benefit, while others had varying degrees of benefit, with none rated as having a negative benefit.
$100 - $150
Hourly Rate
Can velmanase alfa be the next widespread potential therapy for alpha-mannosidosis?
London, UK
2 hours ago
1 Received
- Alpha-mannosidosis (AM) is a rare genetic disorder caused by low levels of the enzyme alpha-mannosidase, leading to symptoms like immunodeficiency, hearing loss, and intellectual disability.
- The disease has various clinical subtypes and is often hard to diagnose early because children may appear normal at birth, highlighting the need for awareness and improved diagnostic processes.
- Treatment options include enzyme replacement therapy with velmanase alfa, which has shown positive clinical effects, but the need for further large-scale studies is crucial to assess its long-term safety and effectiveness.
$100 - $150
Hourly Rate
Diagnosis of alpha-Mannosidosis: Practical approaches to reducing diagnostic delays in this ultra-rare disease.
London, UK
2 hours ago
1 Received
- Alpha-mannosidosis is a rare lysosomal disease caused by mutations in the MAN2B1 gene, leading to a buildup of harmful substances in cells and presenting symptoms similar to other lysosomal diseases, which can delay diagnosis.
- Treatment options were previously limited to hematopoietic stem cell transplantation, but a new enzyme replacement therapy called velmanase alfa has been approved, effective for non-neurological symptoms and improving quality of life, especially in children.
- Raising awareness among patients, families, and healthcare providers through education, disease registries, and advocacy is vital for early diagnosis and treatment to enhance the lives of those affected by alpha-mannosidosis.
$100 - $150
Hourly Rate
Extended long-term efficacy and safety of velmanase alfa treatment up to 12 years in patients with alpha-mannosidosis.
London, UK
2 hours ago
1 Received
- Enzyme replacement therapy (ERT) with velmanase alfa demonstrated effectiveness and safety for up to 12 years in patients with alpha-mannosidosis based on a pooled analysis from two multicenter trials.
- In pediatric patients, improvements in six-minute walk test (6MWT) and stair climb test (3MSCT) were observed, while adult patients showed stabilization or slight decline in performance.
- The treatment resulted in sustained clearance of serum oligosaccharides and increased serum immunoglobulin G (IgG) levels, with most adverse events being mild to moderate in severity, indicating that velmanase alfa is generally well-tolerated.
$100 - $150
Hourly Rate