Digital extensor muscle aplasia-polyneuropathy

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ORPHA:2926OMIM:207740Q87.8
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1FDA treatments8Treatment centers

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Overview

Digital extensor muscle aplasia-polyneuropathy is an extremely rare genetic condition that combines two main problems: the absence (aplasia) of certain muscles in the hands or feet that help straighten the fingers or toes (called digital extensor muscles), along with damage to multiple nerves throughout the body (polyneuropathy). Because the extensor muscles are missing or underdeveloped, affected individuals may have difficulty straightening their fingers or toes, leading to a claw-like hand or foot posture. The polyneuropathy component causes additional nerve-related symptoms such as numbness, tingling, weakness, and sometimes pain in the arms and legs. These nerve problems can worsen over time and may affect balance and coordination. This condition is classified under congenital malformation syndromes (ICD-10 code Q87.8), meaning it is present from birth. Because it is so rare, there is very limited information about its full range of symptoms, natural history, and optimal treatment. Management is primarily supportive and focuses on maintaining hand and foot function through physical therapy, occupational therapy, and sometimes orthopedic interventions such as splints or braces. Treatment of the polyneuropathy may include pain management and medications to support nerve health. There are currently no specific curative treatments or FDA-approved therapies for this condition. Patients benefit from a multidisciplinary care team that includes neurologists, orthopedic specialists, and rehabilitation professionals.

Also known as:

Key symptoms:

Missing or underdeveloped muscles that straighten the fingersInability to fully extend the fingers or toesClaw-like hand or foot postureNumbness or tingling in hands and feetMuscle weakness in the arms and legsReduced grip strengthDifficulty with fine motor tasks like buttoning clothesPain or burning sensations in the limbsLoss of balance or coordinationReduced reflexesMuscle wasting in the hands or feet

Clinical phenotype terms (9)— hover any for plain English
Aplasia/Hypoplasia involving the skeletal musculatureHP:0001460Abnormal nerve conduction velocityHP:0040129Heat intoleranceHP:0002046Abnormality of temperature regulationHP:0004370
Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
Jul 1983

Panhematin: FDA approved

Digital extensor muscle aplasia-polyneuropathy

FDAcompleted

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

Panhematin

Hemin· Abbott LaboratoriesOrphan Drug

No actively recruiting trials found for Digital extensor muscle aplasia-polyneuropathy at this time.

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

Search ClinicalTrials.gov ↗Join the Digital extensor muscle aplasia-polyneuropathy community →

No specialists are currently listed for Digital extensor muscle aplasia-polyneuropathy.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Digital extensor muscle aplasia-polyneuropathy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Digital extensor muscle aplasia-polyneuropathy

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What is the expected progression of the nerve damage in my case?,Are there any genetic tests that could help identify the cause of this condition?,What therapies or exercises can help me maintain the best possible hand and foot function?,Would surgical options like tendon transfers be helpful for my situation?,What medications can help manage nerve pain or other symptoms?,Are there any clinical trials or research studies I could participate in?,Should my family members be evaluated for this condition?

Common questions about Digital extensor muscle aplasia-polyneuropathy

What is Digital extensor muscle aplasia-polyneuropathy?

Digital extensor muscle aplasia-polyneuropathy is an extremely rare genetic condition that combines two main problems: the absence (aplasia) of certain muscles in the hands or feet that help straighten the fingers or toes (called digital extensor muscles), along with damage to multiple nerves throughout the body (polyneuropathy). Because the extensor muscles are missing or underdeveloped, affected individuals may have difficulty straightening their fingers or toes, leading to a claw-like hand or foot posture. The polyneuropathy component causes additional nerve-related symptoms such as numbnes

At what age does Digital extensor muscle aplasia-polyneuropathy typically begin?

Typical onset of Digital extensor muscle aplasia-polyneuropathy is neonatal. Age of onset can vary across affected individuals.

What treatment and support options exist for Digital extensor muscle aplasia-polyneuropathy?

1 patient support program are currently tracked on UniteRare for Digital extensor muscle aplasia-polyneuropathy. See the treatments and support programs sections for copay assistance, eligibility, and contact details.