OBSOLETE: Myostatin-related muscle hypertrophy

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ORPHA:275534
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8Treatment centers

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What is OBSOLETE: Myostatin-related muscle hypertrophy?

Myostatin-related muscle hypertrophy (also known as myostatin-related muscular hypertrophy or muscle hypertrophy syndrome) is a rare genetic condition characterized by significantly increased skeletal muscle mass and reduced body fat. This condition results from mutations in the MSTN gene, which encodes myostatin, a protein that normally acts as a negative regulator of muscle growth. When myostatin function is reduced or absent, muscles grow substantially larger than normal. This entry in Orphanet is marked as OBSOLETE, meaning it may have been reclassified or merged with another entry. Affected individuals typically present with noticeably increased muscle bulk apparent from early childhood, along with increased muscular strength. Importantly, this condition is generally not associated with adverse health effects or medical complications — it primarily manifests as a physical trait of enhanced musculature rather than a debilitating disease. Subcutaneous fat is typically reduced. Cardiac muscle does not appear to be significantly affected based on reported cases. Very few confirmed human cases have been described in the medical literature, making this an exceptionally rare condition. The most well-known case involved a German infant identified in 2004 who displayed extraordinary muscle development at birth. No specific treatment is required or typically pursued, as the condition does not cause functional impairment. Research into the myostatin pathway has generated significant interest for potential therapeutic applications in muscle-wasting diseases such as muscular dystrophies and sarcopenia.

Inheritance
Autosomal recessive
Passed on when both parents carry the same gene change; often skips generations
Age of Onset
Neonatal
Begins at or shortly after birth (first 4 weeks)
Orphanet ↗NORD ↗

Treatments

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

No FDA-approved treatments are currently listed for OBSOLETE: Myostatin-related muscle hypertrophy.

View clinical trials →

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

No actively recruiting trials found for OBSOLETE: Myostatin-related muscle hypertrophy at this time.

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

Search ClinicalTrials.gov ↗Join the OBSOLETE: Myostatin-related muscle hypertrophy 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: Myostatin-related muscle hypertrophy.

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: Myostatin-related muscle hypertrophy.

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Latest news about OBSOLETE: Myostatin-related muscle hypertrophy

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

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Common questions about OBSOLETE: Myostatin-related muscle hypertrophy

What is OBSOLETE: Myostatin-related muscle hypertrophy?

Myostatin-related muscle hypertrophy (also known as myostatin-related muscular hypertrophy or muscle hypertrophy syndrome) is a rare genetic condition characterized by significantly increased skeletal muscle mass and reduced body fat. This condition results from mutations in the MSTN gene, which encodes myostatin, a protein that normally acts as a negative regulator of muscle growth. When myostatin function is reduced or absent, muscles grow substantially larger than normal. This entry in Orphanet is marked as OBSOLETE, meaning it may have been reclassified or merged with another entry. Affec

How is OBSOLETE: Myostatin-related muscle hypertrophy inherited?

OBSOLETE: Myostatin-related muscle hypertrophy follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does OBSOLETE: Myostatin-related muscle hypertrophy typically begin?

Typical onset of OBSOLETE: Myostatin-related muscle hypertrophy is neonatal. Age of onset can vary across affected individuals.

Frequently asked questions about OBSOLETE: Myostatin-related muscle hypertrophy

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

  1. What is OBSOLETE: Myostatin-related muscle hypertrophy?

    OBSOLETE: Myostatin-related muscle hypertrophy is a rare disease catalogued in international rare-disease ontologies (Orphanet ORPHA:275534). It is typically inherited as autosomal recessive. Age of onset is generally neonatal. For verified primary sources, see the UniteRare OBSOLETE: Myostatin-related muscle hypertrophy page.

  2. How is OBSOLETE: Myostatin-related muscle hypertrophy inherited?

    OBSOLETE: Myostatin-related muscle hypertrophy follows autosomal recessive 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: Myostatin-related muscle hypertrophy?

    Approved treatments for OBSOLETE: Myostatin-related muscle hypertrophy 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: Myostatin-related muscle hypertrophy?

    Active clinical trials for OBSOLETE: Myostatin-related muscle hypertrophy 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: Myostatin-related muscle hypertrophy?

    Verified OBSOLETE: Myostatin-related muscle hypertrophy 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: Myostatin-related muscle hypertrophy page for complete clinical details, sources, and verified-specialist listings.

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