ORPHA:217093
Mucopolysaccharidosis type 2, attenuated form
Also known as: Hunter syndrome type B · Iduronate 2-sulfatase deficiency type B · MPS2B · MPSIIB · Mucopolysaccharidosis type 2, non-neuropathic form · Mucopolysaccharidosis type 2, slowly progressive form · Mucopolysaccharidosis type 2B · Mucopolysaccharidosis type II, attenuated form · Mucopolysaccharidosis type IIB
Publications
14
24.4th percentile
Trials
0
Interventional, condition-specific
Researchers
87
Distinct authors in sample
Gene link
—
Readiness
1/6
Stages with a signal
Clinical definition (Orphanet)
Mucopolysaccharidosis type 2, attenuated form (MPS2att), the less severe form of MPS2, leads to a massive accumulation of glycosaminoglycans and a wide variety of symptoms including distinctive facies, short stature, cardiorespiratory and skeletal findings. It is differentiated from mucopolysaccharidosis type 2, severe form by the absence of cognitive decline.
How rare: How common this is has not been clearly measured.
Cross-references
Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.
- MONDO:0016316
- UMLS:C5679815
Additional Mondo synonyms (5)
iduronate 2-sulfatase deficiency type B · mucopolysaccharidosis type 2, attenuated form · mucopolysaccharidosis type 2B · mucopolysaccharidosis type II, attenuated form · mucopolysaccharidosis type IIB
Research stages
Trial readiness signals
Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”
1/6 stages with a signal
Research-stage checklist from open sources (GenCC, literature, Monarch when enriched, ClinicalTrials.gov). Not a prognosis or care recommendation.
- Gene identifiedNot found
No GenCC disease–gene assertion in this build
- LiteraturePresent
14 matched papers (7 in last 10 years) Source
- Phenotype characterisedNot found
No HPO disease–phenotype associations via Monarch for these Mondo IDs
- Animal modelNot found
No Alliance genotype “model of” associations via Monarch for these Mondo IDs
- Orphan designationNot found
No FDA or EMA orphan-drug designation matched this disease via UMLS or preferred name Source
- Interventional trialNot found
No matched interventional trial under our ClinicalTrials.gov rules
Biology
Genes and phenotypes
Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.
Do we know what causes it?
Not yet — the cause hasn't been pinned down in GenCC.
No strong gene–disease assertion joined for this Orphanet entity.
Phenotypes (Monarch / HPO)
None returned for this Mondo ID. That often means “not linked under this ID,” not “no clinical features.”
Animal models (Monarch / Alliance)
None returned for this Mondo ID. Empty here is not proof that no model organism work exists under another name or gene.
Monarch fetch 2026-07-29
Therapies
Designations, candidates, and chemicals
FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.
Orphan designation (FDA · EMA)
No designation matched this disease via UMLS or preferred name on the FDA OOPD mirror or EMA orphan register. Absence here is not proof that none exists under another wording.
Open Targets candidates
No drugs or clinical candidates returned for this Mondo ID on Open Targets.
CTD chemicals (MyDisease.info)
No CTD chemical associations returned for this Mondo ID.
Literature
Is anyone studying this?
14
14 papers have ever been indexed under this name. For scale, breast cancer has over 700,000. Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.
14 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).
7 in the last 10 years · high confidence · 24.4th percentile (publications denominator)
Phrase hits: 14 · MeSH hits: 0
Who's working on it?
87
Distinct author names in 14 sampled papers — named people below.
Who's working on it?
No interventional trial matched this name; these authors publish on it in the sampled literature — a practical starting point for contact.
- 01Nixon RA3 papers · 2024
Center for Dementia Research, Nathan Kline Institute for Psychiatric Research, Orangeburg, NY 10962, USA. nixon@nki.rfmh.org
Papers in Europe PMC - 02Ascensao J1 paper · 1994Papers in Europe PMC
- 03Bainbridge M1 paper · 2024
Rady Children's Institute for Genomic Medicine, San Diego, CA 92123, USA; Rady Children's Hospital, San Diego, CA 92123, USA.
Papers in Europe PMC - 04Baker M1 paper · 2024
Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, WI 53706, USA.
Papers in Europe PMC - 05Batalov S1 paper · 2024
Rady Children's Institute for Genomic Medicine, San Diego, CA 92123, USA; Rady Children's Hospital, San Diego, CA 92123, USA.
Papers in Europe PMC - 06Bergstrom SK1 paper · 1994
Jean Marie Colbert Bone Marrow Transplant Unit, University of Connecticut Health Center, Department of Pediatrics, Farmington 06030.
Papers in Europe PMC - 07Blincow E1 paper · 2024
Rady Children's Institute for Genomic Medicine, San Diego, CA 92123, USA; Rady Children's Hospital, San Diego, CA 92123, USA.
Papers in Europe PMC - 08Cao B1 paper · 2024
Rady Children's Institute for Genomic Medicine, San Diego, CA 92123, USA; Rady Children's Hospital, San Diego, CA 92123, USA.
Papers in Europe PMC - 09Carson D1 paper · 1989Papers in Europe PMC
- 10Caylor S1 paper · 2024
Rady Children's Institute for Genomic Medicine, San Diego, CA 92123, USA; Rady Children's Hospital, San Diego, CA 92123, USA.
Papers in Europe PMC
Clinical research
Is a treatment being tested?
0
interventional trials for this specific condition
No interventional trial testing a treatment matched this specific condition name on ClinicalTrials.gov (observational studies and pan-disease registries are listed separately when present).
Data as of 11 September 2026 · last trial check 11 September 2026
No matched interventional trials. This is true for 77.2% of diseases in the trials denominator (5501 of 7126). Here are the researchers publishing on it.
high confidence · 38.6th percentile (trials denominator)
Recruiting interventional trials
From the matched ClinicalTrials.gov set
No interventional trial testing a treatment was found for this specific condition name on ClinicalTrials.gov.
Broader category mucopolysaccharidosis type 2 also has no matched interventional trial. See who's working on it — people publishing on this disease are often the practical next contact when no trial is listed.
Broader category: mucopolysaccharidosis type 2
0
Interventional trials for the parent category, exclusive of NCT IDs already counted above. Eligibility for this subtype is not guaranteed.
Parent-category matching found a broader label but no interventional trials under it. How we count trials.
Other registries (secondary)
Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.
raw 7 · after dedupe 7 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 7 · dropped 0 · fetched 2026-07-30
Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri
No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).
Uncertain / not reviewed (7)
- isrctn·ISRCTN46766641·No longer recruiting·A Phase I randomized, open-label pharmacokinetic comparability study comparing pre- and post-change teclistamab in participants with relapsed/refractory multiple myeloma
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN16919215·No longer recruiting·Hydrotherapy in mucopolysaccharidosis II
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN13106662·Recruiting·Will the medication modafinil reduce post-stroke fatigue and improve quality of life of patients?
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN12220131·No longer recruiting·A study comparing talquetamab in combination with daratumumab or in combination with daratumumab and pomalidomide versus daratumumab in combination with pomalidomide and dexamethasone in participants with multiple myeloma that returns after treatment or is resistant to treatment
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN11652897·No longer recruiting·Study to determine the effectiveness and safety of DNL310 vs idursulfase in pediatric participants with neuronopathic or non-neuronopathic Hunter Syndrome
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN11577984·No longer recruiting·A study on the safety and effects of the drug DMT in healthy smoking individuals
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN42835524·No longer recruiting·Phase I Single- and Multiple-Ascending Dose Trial of EVX-101
skipped — LLM skipped (--skip-llm)
Where to find support
Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.
Orphanet entry for Mucopolysaccharidosis type 2, attenuated form — check Associations / patient organisations on that page.
India — NPRD
Last verified 2026-07-26Likely covered — the policy lists Mucopolysaccharidosis type II (Hunter) as a category (Group 3), and this condition is a form of it. Confirm eligibility with a Centre of Excellence.
Group 3 — high-cost / lifelong therapy with careful selection
Up to ₹50 lakh per patient
Financial support at notified Centres of Excellence is the figure commonly cited in recent MoHFW/PIB statements. Many Group 3 patients also use the MoHFW voluntary-contribution / crowdfunding portal.
Eligibility and patient selection rules change. Verify with a CoE; the crowdfunding portal is a separate mechanism from CoE funding. Verify
Centres of Excellence (15)
- All India Institute of Medical Sciences (AIIMS) — New Delhi, Delhi
- Maulana Azad Medical College — New Delhi, Delhi
- Sanjay Gandhi Post Graduate Institute of Medical Sciences — Lucknow, Uttar Pradesh
- Post Graduate Institute of Medical Education and Research (PGIMER) — Chandigarh, Chandigarh
- Centre for DNA Fingerprinting & Diagnostics with Nizam’s Institute of Medical Sciences — Hyderabad, Telangana
- King Edward Memorial Hospital — Mumbai, Maharashtra
- Institute of Post-Graduate Medical Education and Research (IPGMER) — Kolkata, West Bengal
- Centre for Human Genetics with Indira Gandhi Hospital — Bengaluru, Karnataka
- Institute of Child Health and Hospital for Children (ICH & HC) — Chennai, Tamil Nadu
- All India Institute of Medical Sciences (AIIMS) — Jodhpur, Rajasthan
- Sree Avittam Thirunal Hospital (SAT), Government Medical College — Thiruvananthapuram, Kerala
- All India Institute of Medical Sciences (AIIMS) — Bhopal, Madhya Pradesh
- Regional Institute of Medical Sciences (RIMS) — Imphal, Manipur
- All India Institute of Medical Sciences (AIIMS) — Patna, Bihar
- Assam Medical College & Hospital — Dibrugarh, Assam
Voluntary contributions / crowdfunding (separate from CoE funding): https://rarediseases.mohfw.gov.in/
Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.
How we counted this
Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.
"Mucopolysaccharidosis type 2, attenuated form" OR "Hunter syndrome type B" OR "Iduronate 2-sulfatase deficiency type B" OR "MPS2B" OR "MPSIIB" OR "Mucopolysaccharidosis type 2, non-neuropathic form" OR "Mucopolysaccharidosis type 2, slowly progressive form" OR "Mucopolysaccharidosis type 2B" OR "Mucopolysaccharidosis type II, attenuated form" OR "Mucopolysaccharidosis type IIB"
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Mucopolysaccharidosis type 2, attenuated form" OR "Hunter syndrome type B" OR "Iduronate 2-sulfatase deficiency type B" OR "MPS2B" OR "MPSIIB" OR "Mucopolysaccharidosis type 2, non-neuropathic form" OR "Mucopolysaccharidosis type 2, slowly progressive form" OR "Mucopolysaccharidosis type 2B" OR "Mucopolysaccharidosis type II, attenuated form" OR "Mucopolysaccharidosis type IIB"
Study-type breakdown: 0 interventional · 0 observational · 0 expanded access. Only interventional studies enter the trial headline.
Parent-category trials query:
"mucopolysaccharidosis type 2"
Query health: ok — strategies attempted: phrase; with hits: phrase
Run this search on ClinicalTrials.gov
Confidence reasoning
- Preferred label is multi-word and distinctive
- No synonyms dropped by stoplist
- No label/synonym collisions with other diseases in this corpus
Ingested 2026-07-27T09:45:50.012Z