Rivfloza (nedosiran)
Research Synopsis
- * The drug works by inhibiting the hepatic lactate dehydrogenase enzyme, significantly reducing urinary oxalate levels in patients with PH type 1 and PH type 2, demonstrating a strong safety profile with low rates of adverse effects.
- * Clinical trials have shown that nedosiran can lead to normal or near-normal oxalate excretion in nearly half of the treated patients, indicating its efficacy in managing the condition.
- * Recent studies suggest that nedosiran has the potential to postpone the need for more invasive treatment options, like combined kidney and liver transplants, especially for patients with primary hyperoxaluria type 1 who often face severe health challenges.
- * The FDA approval on September 29, 2023, marked a significant milestone in treatment options for PH, particularly benefiting children aged nine and older and adults with relatively preserved kidney function.
- * Nedosiran's success contributes to the growing field of RNA interference therapeutics, paving the way for future advancements targeting other rare diseases linked to abnormal enzyme functions.
- * Ongoing research continues to assess the long-term efficacy and safety of nedosiran, with investigations into its effects in patients with advanced chronic kidney disease and its potential for other subtypes of primary hyperoxaluria.
- * The therapeutic landscape for primary hyperoxaluria has rapidly evolved, emphasizing the importance of RNA-based treatments like nedosiran in improving patient outcomes and quality of life.
- * As more clinical data becomes available, there is potential for nedosiran to play a vital role in changing management strategies for primary hyperoxaluria, minimizing the necessity for transplantation.
Rivfloza (nedosiran)
Hepatic Lactate Dehydrogenase A: An RNA Interference Target for the Treatment of All Known Types of Primary Hyperoxaluria.
London, UK
2 hours ago
1 Received
- Primary hyperoxaluria (PH) is a group of rare genetic disorders that cause excessive oxalate production, leading to kidney damage, and RNA interference (RNAi) shows promise as a treatment for this condition.
- * Nonclinical studies in animals indicated that RNAi specifically reduces urinary oxalate levels without affecting other tissues, such as muscle.
- * Phase I clinical trial results for nedosiran, an RNAi therapy, showed no negative effects on muscle health or function, supporting the safety of this treatment for PH.
$100 - $150
Hourly Rate
The growth of siRNA-based therapeutics: Updated clinical studies.
London, UK
2 hours ago
1 Received
- * Most late-stage siRNA drugs target rare diseases, but some, like inclisiran, aim to treat more common conditions such as high cholesterol even in patients not responding to traditional treatments.
- * The review discusses how siRNA works, challenges in delivering it effectively, and the modifications and delivery methods being used, while also profiling both approved siRNA drugs and those in Phase 3 trials, including their mechanisms and clinical progress.
$100 - $150
Hourly Rate
Nedosiran Dramatically Reduces Serum Oxalate in Dialysis-Dependent Primary Hyperoxaluria 1: A Compassionate Use Case Report.
London, UK
2 hours ago
1 Received
- Primary hyperoxaluria 1 (PH1) is a serious condition characterized by kidney stones, rapid kidney failure, and buildup of calcium oxalate in the body.
- Treatment often requires a combined kidney and liver transplant due to limited options available.
- In a reported case, monthly injections of Nedosiran, an RNA interference therapy, significantly lowered plasma oxalate levels and reduced the need for hemodialysis, postponing the need for transplant in the patient.
$100 - $150
Hourly Rate
Safety, pharmacodynamics, and exposure-response modeling results from a first-in-human phase 1 study of nedosiran (PHYOX1) in primary hyperoxaluria.
London, UK
2 hours ago
1 Received
- Primary hyperoxaluria (PH) is a rare genetic disorder leading to excessive oxalate production due to faulty liver metabolism, which can cause kidney stones and other complications.
- Nedosiran is a new treatment that uses RNA interference to block an enzyme involved in oxalate production, and was tested in a study with healthy individuals and PH patients.
- Results showed that nedosiran was safe and led to a significant reduction in urinary oxalate levels; almost one-third of PH patients achieved normal oxalate excretion after treatment.
$100 - $150
Hourly Rate
Primary hyperoxaluria type 1: novel therapies at a glance.
London, UK
2 hours ago
1 Received
- Primary hyperoxaluria type 1 (PH1) is a serious genetic disorder affecting oxalate metabolism due to a mutation in the AGT enzyme, leading to kidney failure and requiring treatments like hydration, medications, and transplants.
- Recent advancements in treatment include RNA interference (RNAi) therapies, such as lumasiran and nedosiran, which aim to reduce oxalate production by targeting specific liver enzymes.
- Lumasiran has been approved for PH1, showing promise in reducing urinary oxalate levels, while ongoing trials for nedosiran seek further evidence of its long-term effectiveness and safety.
$100 - $150
Hourly Rate
Treatment of primary hyperoxaluria type 1.
London, UK
2 hours ago
1 Received
- Supportive treatment for primary hyperoxaluria type 1 (PH1) includes high fluid intake and medications like pyridoxine for some genotypes, which can significantly reduce urinary oxalate levels.
- The first RNA interference therapy was approved in 2020, promising to improve treatment strategies and long-term outcomes for PH1 patients suffering from kidney impairment and the risk of oxalosis.
- Liver transplantation can effectively cure PH1 by restoring enzyme function and preventing further oxalate production, often paired with kidney transplantation, depending on patient-specific factors.
$100 - $150
Hourly Rate
Therapeutic RNA interference: A novel approach to the treatment of primary hyperoxaluria.
London, UK
2 hours ago
1 Received
- * The article discusses the cellular RNAi system and outlines how RNAi therapies are designed and delivered, highlighting a specific ultra-rare disease called primary hyperoxaluria, which can lead to severe kidney issues.
- * Two new RNAi therapies, nedosiran and lumasiran, are presented as treatments that target specific enzymes in the liver to decrease harmful oxalate production in patients with primary hyperoxaluria, along with a look at future improvements in RNAi approaches.
$100 - $150
Hourly Rate
New therapeutics for primary hyperoxaluria type 1.
London, UK
2 hours ago
1 Received
- Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder leading to excess oxalate production, which can cause severe kidney issues.
- Recent advancements in therapies, particularly RNA interference (RNAi) treatments like Lumasiran and Nedosiran, show promise in lowering oxalate levels in patients.
- Other potential treatments and genetic editing tools are in development, aiming to reduce the necessity for combined kidney and liver transplants and improve patient outcomes.
$100 - $150
Hourly Rate
Progress with RNA Interference for the Treatment of Primary Hyperoxaluria.
London, UK
2 hours ago
1 Received
- Recent FDA-approved drugs employing RNA interference have been developed to treat liver-specific diseases, particularly benefiting conditions like primary hyperoxaluria type 1.* -
- Primary hyperoxaluria type 1, a disorder caused by enzyme mutations leading to high oxalate levels, historically had limited treatment options, mainly supportive care and transplants.* -
- The approval of the RNA interference drug lumasiran and the potential approval of nedosiran represent a significant advancement in treatment, potentially changing disease outcomes despite some limitations for other types of hyperoxaluria.*
$100 - $150
Hourly Rate
Improving Treatment Options for Primary Hyperoxaluria.
London, UK
2 hours ago
1 Received
- Primary hyperoxalurias are rare liver disorders that cause increased oxalate production, leading to kidney stones and potential kidney failure, especially in type 1.
- Current treatments include hyperhydration, alkaline citrate, and vitamin B therapy, which can help some patients by lowering urinary oxalate levels.
- New treatments like RNA interference medications (Lumasiran and Nedosiran) aim to reduce oxalate production and future options may include substrate reduction therapy, stem cell therapy, and gene therapy.
$100 - $150
Hourly Rate
Nedosiran, a Candidate siRNA Drug for the Treatment of Primary Hyperoxaluria: Design, Development, and Clinical Studies.
London, UK
2 hours ago
1 Received
- There is a critical need for effective drug therapies for the genetic disorder primary hyperoxaluria (PH), which includes three subtypes: PH1, PH2, and PH3, characterized by oxalate accumulation leading to kidney stones.
- Currently, nedosiran, an siRNA-based drug, is being developed to treat all three PH types, and it has shown promise in clinical trials by significantly reducing urinary oxalate levels in patients with PH1.
- Clinical trials have also indicated that nedosiran has a favorable safety profile, with mostly mild injection site reactions and no severe adverse events reported, making it a strong candidate for treating PH.
$100 - $150
Hourly Rate
Kidney Organoids Generated Using an Allelic Series of NPHS2 Point Variants Reveal Distinct Intracellular Podocin Mistrafficking.
London, UK
2 hours ago
1 Received
- NPHS2 variants are the leading cause of steroid-resistant nephrotic syndrome in children, with specific missense variants affecting protein trafficking of PODOCIN in podocytes, based on studies using nonpodocyte cell lines.
- Researchers created human iPSC lines with pathogenic NPHS2 variants and differentiated them into kidney organoids to observe the effects on PODOCIN expression and subcellular localization.
- The study found that NPHS2 variants altered PODOCIN localization in podocytes, with some variants causing accumulation in the endoplasmic reticulum or Golgi, leading to insights into how these mutations disrupt podocyte function and contribute to the disease.
$100 - $150
Hourly Rate
PHYOX2: a pivotal randomized study of nedosiran in primary hyperoxaluria type 1 or 2.
London, UK
2 hours ago
1 Received
- Nedosiran is an experimental RNA interference treatment aimed at reducing the production of oxalate by inhibiting an enzyme involved in its synthesis, targeting primary hyperoxaluria (PH) patients.
- In a six-month study, participants receiving nedosiran experienced a significant reduction in 24-hour urinary oxalate levels compared to those given a placebo, with half of the nedosiran group achieving normal or near-normal excretion levels.
- The treatment was well tolerated with low rates of mild injection-site reactions, showing promising results in lowering oxalate levels in patients with PH1, while the effects in patients with PH2 were less consistent.
$100 - $150
Hourly Rate
Nedosiran in primary hyperoxaluria subtype 3: results from a phase I, single-dose study (PHYOX4).
London, UK
2 hours ago
1 Received
- Nedosiran is an RNA interference agent designed to lower oxalate production in patients with primary hyperoxaluria subtype 3 (PH3) by targeting a specific liver enzyme.
- In a phase I clinical trial, researchers tested the safety and effectiveness of a single dose of nedosiran compared to a placebo in six participants, finding it well-tolerated with no major safety issues.
- Although the primary measure of effectiveness wasn’t fully met, nedosiran showed a reduction in urinary oxalate excretion, suggesting it may be a promising treatment option for PH3 patients who currently lack effective therapies.*
$100 - $150
Hourly Rate
Correction to: Nedosiran in primary hyperoxaluria subtype 3: results from a phase I, single-dose study (PHYOX4).
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
A Case Report of Kidney-Only Transplantation in Primary Hyperoxaluria Type 1: A Novel Approach with the Use of Nedosiran.
London, UK
2 hours ago
1 Received
- Primary hyperoxalurias (PHs) are kidney disorders leading to stones, nephrocalcinosis, and chronic kidney disease, resulting in excessive oxalate levels and potential tissue damage as kidney function declines.
- Current treatment for primary hyperoxaluria type 1 (PH1) often requires combined liver and kidney transplantation, but kidney-only transplants have shown success in select patients treated with pyridoxine or the RNA therapy lumasiran.
- This article reviews PH1, explores small interfering RNA therapies like lumasiran and nedosiran, and discusses cases of kidney-only transplants in PH1 patients, highlighting innovative management options for advanced kidney disease.
$100 - $150
Hourly Rate
Safety, Pharmacokinetics, and Exposure-Response Modeling of Nedosiran in Participants With Severe Chronic Kidney Disease.
London, UK
2 hours ago
1 Received
- Nedosiran is an investigational RNA-interference therapy aimed at treating primary hyperoxaluria (PH), specifically focusing on its pharmacokinetics in individuals with normal kidney function versus those with advanced chronic kidney disease (CKD) stages 4/5.* -
- A Phase 1 study involved 34 participants receiving a single dose of nedosiran, revealing that those with CKD Stages 4/5 experienced roughly double the medication exposure compared to those with normal kidney function, but hemodialysis timing did not significantly affect this.* -
- Modeling results recommend lower doses of nedosiran for patients with PH Subtype 1 and CKD Stages 4/5 while indicating that
$100 - $150
Hourly Rate
Nedosiran: First Approval.
London, UK
2 hours ago
1 Received
- Nedosiran (RIVFLOZA™) is a monthly subcutaneous siRNA therapy being developed by Dicerna Pharmaceuticals for treating primary hyperoxaluria (PH), a condition involving excessive oxalate production.
- It works by inhibiting the hepatic lactate dehydrogenase (LDH) enzyme to reduce oxalate levels in the body.
- The therapy received its first approval on September 29, 2023, in the USA for use in children aged 9 and older and adults with PH type 1 and relatively preserved kidney function.
$100 - $150
Hourly Rate
Correction to: Nedosiran: First Approval.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
2023 FDA TIDES (Peptides and Oligonucleotides) Harvest.
London, UK
2 hours ago
1 Received
- In 2023, the FDA approved nine TIDES, including four oligonucleotides targeting disorders like ALS, geographic atrophy, primary hyperoxaluria type 1, and hereditary transthyretin-mediated amyloidosis, all with enhanced stability and effectiveness.
- The approved peptides exhibit a variety of structures (linear, cyclic, and lipopeptides) and applications, including the first orphan drug designation for a peptide-based chemokine antagonist.
- Notably, a peptide-based treatment was approved for core symptoms of Rett syndrome, with the analysis of TIDES focusing on their chemical structure, medical targets, and potential side effects.
$100 - $150
Hourly Rate