PulseExploreJournal ClubDebatesTrendingResearchersJournals
Instagram
HomeExploreJournal ClubTrending
Synapse
⌘+K
Synapse

Therapeutic Advances in Rare Disease

  • h-index: 10
  • Total citations: 322
  • Total papers: 43
  • Impact factor: 2.727272727272727

Homepage: https://us.sagepub.com/en-us/nam/therapeutic-advances-in-rare-disease/journal203681

Therapeutic Advances in Rare Disease

Journal
Visit website →
H-Index = 10
Citations = 322

Papers

Evidence

Recent papers in Therapeutic Advances in Rare Disease

  1. Endothelial–mitochondrial coupling in mitochondrial disease: A systematic review and quantitative synthesis of vascular, biochemical, and oxidative bioenergetic dysfunctionTherapeutic Advances in Rare Disease · Tue,
  2. Evaluation of muscle microvascular perfusion in primary mitochondrial disease by contrast-enhanced ultrasound: Feasibility studyTherapeutic Advances in Rare Disease · Tue,
  3. First bone marrow transplantation for mucopolysaccharidosis type I in Vietnam: a case reportTherapeutic Advances in Rare Disease · Sat,
  4. Interim analysis of an international multi-site prospective natural history study evaluating the clinical presentation and progression of Leigh syndrome spectrum disordersTherapeutic Advances in Rare Disease · Sat,
  5. Current therapeutic landscape of β-thalassemia: focus on gene therapyTherapeutic Advances in Rare Disease · Sun,
  6. Could an outcome-based agreement be operationalized using real-world data from the Canadian Neuromuscular Disease Registry? Perspectives from an expert-led assessment in spinal muscular atrophyTherapeutic Advances in Rare Disease · Sun,
  7. Reliability assessment using the test–retest method and minimal important changes in the Adult Fabry Disease Quality of Life ScaleTherapeutic Advances in Rare Disease · Sun,
  8. Challenges and opportunities for the use of telehealth in rare disease diagnosis, treatment, research, and education: key opinion leader interviews by the IRDiRC telehealth task forceTherapeutic Advances in Rare Disease · Sun,
  9. The PBC Ireland patient registry: study protocol for a national platform on primary biliary cholangitisTherapeutic Advances in Rare Disease · Sun,
  10. Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe diseaseTherapeutic Advances in Rare Disease · Sun,

About Therapeutic Advances in Rare Disease

Therapeutic Advances in Rare Disease carries 43 indexed papers, 322 citations, h-index 10 in Synapse's enriched corpus. Each paper indexed here includes structured clinical evidence extraction (PICO), methodology classification with level of evidence, and links to related guideline recommendations where applicable.

On Synapse's credibility score (derived from SJR ranking and cross-citation patterns), Therapeutic Advances in Rare Disease sits among the highest-credibility cardiovascular journals.

The journal's official homepage is at https://us.sagepub.com/en-us/nam/therapeutic-advances-in-rare-disease/journal203681.

Use the feed below to follow new Therapeutic Advances in Rare Disease publications with AI-enriched analysis — each paper is decomposed into study design, population, intervention, comparator, primary outcome, and key finding, with the underlying evidence traceable to the primary source.

Deterministic synthesis from Synapse's enriched records — 111 words.