Safety and Efficacy of Genistein in Sanfilippo Syndrome - A SystematicReview
Bibliographic record
Abstract
Background: Sanfilippo syndrome, commonly known as mucopolysaccharidosis type III (MPS III), is a rare autosomal recessive lysosomal storage disease that primarily affects the brain and spinal cord. It is caused by a deficiency of enzymes involved in the catabolism of glycosaminoglycan (GAG) and heparin sulfate (HS). Although Genistein has been presented as a potential therapeutic, its safety and efficacy for the treatment of Sanfilippo syndrome are not well established. Objective: This systematic review aims to evaluate the safety and efficacy of Genistein with a primary endpoint of providing an inference whether the medication is producing any improvements when administered in patients suffering from Sanfilippo syndrome. Methods: An intensive computerized literature search was performed according to PRISMA guidelines in major databases such as PubMed, ScienceDirect, Embase, Science Citation Index Expanded, The Cochrane Library, and Web of Science until 30 April 2022. Studies evaluating the efficacy and safety of genistein in patients with Sanfilippo Syndrome were included. The quality of the included studies was assessed using the New Castle Ottawa Scale. Results: A total of 558 studies were identified in the initial search. After removing duplicates, 25 studies were screened based on title and abstract, of which 06 studies were included in this systematic review. Among them, 02 studies were of high quality and 04 studies were of moderate quality. After administration of genistein, a decrease in urinary GAG levels and HS levels was observed. Conclusion: Based on the limited evidence present in the literature, this systematic review suggests that genistein can be safely used to treat Sanfilippo syndrome as it lowers GAG levels. However, data on the long-term benefit profile of genistein and high-quality evidence are still needed as a conclusive result could not be withdrawn.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.007 | 0.028 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.008 | 0.007 |
| Bibliometrics | 0.008 | 0.007 |
| Science and technology studies | 0.001 | 0.001 |
| Scholarly communication | 0.002 | 0.002 |
| Open science | 0.002 | 0.001 |
| Research integrity | 0.002 | 0.001 |
| Insufficient payload (model declined to judge) | 0.004 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".