OBSOLETE: Cys-loop receptor channelopathy

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ORPHA:98121
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8Treatment centers

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What is OBSOLETE: Cys-loop receptor channelopathy?

Cys-loop receptor channelopathy is an obsolete grouping term previously used in Orphanet (ORPHA:98121) to classify a heterogeneous set of genetic disorders caused by mutations in genes encoding Cys-loop ligand-gated ion channel receptors. The Cys-loop receptor superfamily includes nicotinic acetylcholine receptors (nAChRs), glycine receptors, GABA-A receptors, and serotonin type 3 (5-HT3) receptors. These receptors are critical for fast synaptic transmission in the central and peripheral nervous systems. Mutations in genes encoding subunits of these channels can lead to a variety of neurological conditions, including certain congenital myasthenic syndromes (affecting the neuromuscular junction), hyperekplexia or startle disease (glycine receptor mutations), and some forms of epilepsy (GABA-A receptor or nicotinic receptor mutations). Because this term is classified as obsolete, it is no longer used as a standalone diagnostic entity. Instead, the individual conditions that were grouped under this umbrella — such as autosomal dominant nocturnal frontal lobe epilepsy, hyperekplexia, and various congenital myasthenic syndromes — are now classified and coded separately. Each of these specific disorders has its own distinct genetic basis, inheritance pattern, clinical presentation, and management approach. Patients who may have previously been categorized under this grouping should refer to the specific diagnosis relevant to their condition for accurate clinical information, genetic counseling, and treatment options. Treatments vary widely depending on the specific channelopathy and may include antiepileptic drugs, clonazepam (for hyperekplexia), or acetylcholinesterase inhibitors (for certain myasthenic syndromes).

Inheritance
Variable
Can be inherited in different ways depending on the underlying gene
Age of Onset
Variable
Can begin at different ages, from infancy through adulthood
Orphanet ↗NORD ↗

Treatments

Source: openFDA + DailyMed · NDA / BLA labels with structured indications · refreshed weekly

No FDA-approved treatments are currently listed for OBSOLETE: Cys-loop receptor channelopathy.

View clinical trials →

Source: ClinicalTrials.gov · synced daily · phases, status, and PI names normalized at ingest

No actively recruiting trials found for OBSOLETE: Cys-loop receptor channelopathy at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the OBSOLETE: Cys-loop receptor channelopathy community →

Source: NPI Registry + PubMed · trial PI roles cross-referenced with ClinicalTrials.gov · ranked by match score (publications + PI activity + community signal)

No specialists are currently listed for OBSOLETE: Cys-loop receptor channelopathy.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers

Source: NORD Rare Disease Centers + NIH Undiagnosed Diseases Network (UDN) · centers verified active within last 12 months

🏨 Children's

Children's Hospital Colorado Rare Disease Program

Children's Hospital Colorado

📍 Aurora, CO

👤 Boston Children's Hospital Rare Disease Program

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Boston Children's Hospital Rare Disease Program

Boston Children's Hospital

📍 Boston, MA

👤 Boston Children's Hospital Rare Disease Program

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

🏨 Children's

Ann & Robert H. Lurie Children's Hospital Genetics

Lurie Children's Hospital

📍 Chicago, IL

👤 Boston Children's Hospital Rare Disease Program

🏥 NORD

Cincinnati Children's Hospital Medical Center

Cincinnati Children's

📍 Cincinnati, OH

👤 Boston Children's Hospital Rare Disease Program

🏨 Children's

Nationwide Children's Hospital Rare Disease Center

Nationwide Children's Hospital

📍 Columbus, OH

👤 Boston Children's Hospital Rare Disease Program

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

Travel Grants

No travel grants are currently matched to OBSOLETE: Cys-loop receptor channelopathy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about OBSOLETE: Cys-loop receptor channelopathy

Source: PubMed + NIH RePORTER + openFDA + clinical-journal RSS · last 30 days · disease-tagged at ingest by AI extraction with human QC

No recent news articles for OBSOLETE: Cys-loop receptor channelopathy.

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Common questions about OBSOLETE: Cys-loop receptor channelopathy

What is OBSOLETE: Cys-loop receptor channelopathy?

Cys-loop receptor channelopathy is an obsolete grouping term previously used in Orphanet (ORPHA:98121) to classify a heterogeneous set of genetic disorders caused by mutations in genes encoding Cys-loop ligand-gated ion channel receptors. The Cys-loop receptor superfamily includes nicotinic acetylcholine receptors (nAChRs), glycine receptors, GABA-A receptors, and serotonin type 3 (5-HT3) receptors. These receptors are critical for fast synaptic transmission in the central and peripheral nervous systems. Mutations in genes encoding subunits of these channels can lead to a variety of neurologic

Frequently asked questions about OBSOLETE: Cys-loop receptor channelopathy

Auto-generated from canonical disease facts (Orphanet, OMIM, ClinicalTrials.gov, openFDA, NPPES). Not a substitute for clinical guidance.

  1. What is OBSOLETE: Cys-loop receptor channelopathy?

    OBSOLETE: Cys-loop receptor channelopathy is a rare disease catalogued in international rare-disease ontologies (Orphanet ORPHA:98121). It is typically inherited as variable. Age of onset is generally variable. For verified primary sources, see the UniteRare OBSOLETE: Cys-loop receptor channelopathy page.

  2. How is OBSOLETE: Cys-loop receptor channelopathy inherited?

    OBSOLETE: Cys-loop receptor channelopathy follows variable inheritance. Genetic counseling is recommended for affected families to understand recurrence risk in offspring and the likelihood of unaffected siblings being carriers. Variants in the underlying gene(s) may be identified via clinical genetic testing.

  3. Are there FDA-approved treatments for OBSOLETE: Cys-loop receptor channelopathy?

    Approved treatments for OBSOLETE: Cys-loop receptor channelopathy are tracked from openFDA and DailyMed primary sources. Many rare diseases have no specific FDA-approved therapy; for those, supportive care and management of complications form the basis of clinical care. Orphan-drug-designation status is noted where applicable.

  4. Are there clinical trials for OBSOLETE: Cys-loop receptor channelopathy?

    Active clinical trials for OBSOLETE: Cys-loop receptor channelopathy are tracked daily from ClinicalTrials.gov. Trial availability changes frequently; check the UniteRare trial listings for the current count and recruitment status. Sponsors of rare-disease research often welcome inquiries even when a trial is not actively recruiting at a given moment.

  5. How do I find a specialist for OBSOLETE: Cys-loop receptor channelopathy?

    Verified OBSOLETE: Cys-loop receptor channelopathy specialists are identified through ClinicalTrials.gov principal-investigator records, peer-reviewed publication authorship (via PubMed), and the NPPES NPI registry. NORD-designated Centers of Excellence and NIH-affiliated rare-disease clinics are also tracked. UniteRare's specialist directory is updated continuously as new evidence becomes available.

See full OBSOLETE: Cys-loop receptor channelopathy page for complete clinical details, sources, and verified-specialist listings.

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