Infantile-onset ascending hereditary spastic paralysis

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ORPHA:293168OMIM:607225G12.2
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20Specialists8Treatment centers

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Overview

Infantile-onset ascending hereditary spastic paralysis (IAHSP) is an extremely rare genetic condition that affects the motor neurons — the nerve cells that control voluntary muscle movement. It is sometimes also called infantile-onset ascending hereditary spastic paraplegia. The disease begins in infancy, typically within the first two years of life, and causes progressive stiffness (spasticity) and weakness that starts in the legs and gradually moves upward to affect the arms and eventually the muscles involved in speaking and swallowing. Children with IAHSP usually develop normally at first but then begin to show difficulty with walking and movement. Over time, the stiffness and weakness spread to the upper body. The condition primarily affects the upper motor neurons, which are the nerve cells in the brain and spinal cord that send signals to the muscles. Unlike some related conditions, IAHSP does not typically affect thinking or intelligence. There is currently no cure for IAHSP. Treatment focuses on managing symptoms and maintaining quality of life. This includes physical therapy to preserve mobility and reduce stiffness, medications to manage spasticity, and assistive devices such as braces or wheelchairs as the disease progresses. Speech therapy and feeding support may become necessary as the condition advances. Because the disease is so rare, research is limited, but genetic understanding has improved with the identification of the responsible gene.

Also known as:

Key symptoms:

Progressive leg stiffness (spasticity)Difficulty walking or inability to walkWeakness that starts in the legs and moves upwardStiffness and weakness in the armsDifficulty speaking (dysarthria)Difficulty swallowing (dysphagia)Exaggerated reflexesAbnormal walking patternMuscle wasting over timeLoss of fine motor skills in the handsBreathing difficulties in advanced stagesInvoluntary muscle spasmsProgressive loss of mobility

Clinical phenotype terms (11)— hover any for plain English
Pseudobulbar affectHP:0002193AnarthriaHP:0002425TetraplegiaHP:0002445Spastic tetraplegiaHP:0002510Impaired masticationHP:0005216
Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Infantile-onset ascending hereditary spastic paralysis.

View clinical trials →

No actively recruiting trials found for Infantile-onset ascending hereditary spastic paralysis at this time.

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Search ClinicalTrials.gov ↗Join the Infantile-onset ascending hereditary spastic paralysis community →

Specialists

20 foundView all specialists →
LP
Li Cao, phD
HOUSTON, TX
Specialist
PI on 2 active trials
FP
Filippo M Santorelli, MD PhD
Specialist
PI on 2 active trials
TM
Tim W. Rattay, MD
Specialist
PI on 1 active trial
LM
Laurent Servais, MD
Specialist
PI on 6 active trials1 Infantile-onset ascending hereditary spastic paralysis publication
LP
Leonardo Boccuni, PhD
Specialist
PI on 1 active trial
JG
Joseph Gleeson
SAN DIEGO, CA
Specialist
PI on 1 active trial43 Infantile-onset ascending hereditary spastic paralysis publications
FD
Filippo M Santorelli, Dr.
Specialist
PI on 2 active trials
MP
Marcondes c França Júnior, M.D, PhD
Specialist
PI on 1 active trial
JM
Jakub M Antczak, MD
Krakow, Lesser Poland Voivodeship
Specialist

Rare Disease Specialist

PI on 3 active trials
RD
Rebecca Schüle, PD Dr.
Specialist
PI on 2 active trials
FS
Ferda Selcuk
Specialist
PI on 1 active trial1 Infantile-onset ascending hereditary spastic paralysis publication
JM
Jeffrey Statland, MD
KANSAS CITY, KS
Specialist
PI on 7 active trials
AM
Alan Hand, MD
SUGAR LAND, TX
Specialist
PI on 1 active trial1 Infantile-onset ascending hereditary spastic paralysis publication
LD
Ludger Schöls, Prof. Dr.
Specialist
PI on 3 active trials
AD
A.C.H. Geurts, Prof. Dr.
Specialist
PI on 1 active trial

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 Infantile-onset ascending hereditary spastic paralysis.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

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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.How quickly is this disease likely to progress in my child's case?,What therapies and medications can help manage the spasticity?,Should we consider an intrathecal baclofen pump, and when would that be appropriate?,Are there any clinical trials or research studies we could participate in?,What signs should we watch for that would indicate the disease is affecting breathing or swallowing?,How can we best support my child's development and education?,Should other family members be tested for the ALS2 gene mutation?

Common questions about Infantile-onset ascending hereditary spastic paralysis

What is Infantile-onset ascending hereditary spastic paralysis?

Infantile-onset ascending hereditary spastic paralysis (IAHSP) is an extremely rare genetic condition that affects the motor neurons — the nerve cells that control voluntary muscle movement. It is sometimes also called infantile-onset ascending hereditary spastic paraplegia. The disease begins in infancy, typically within the first two years of life, and causes progressive stiffness (spasticity) and weakness that starts in the legs and gradually moves upward to affect the arms and eventually the muscles involved in speaking and swallowing. Children with IAHSP usually develop normally at first

How is Infantile-onset ascending hereditary spastic paralysis inherited?

Infantile-onset ascending hereditary spastic paralysis follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Infantile-onset ascending hereditary spastic paralysis typically begin?

Typical onset of Infantile-onset ascending hereditary spastic paralysis is infantile. Age of onset can vary across affected individuals.

Which specialists treat Infantile-onset ascending hereditary spastic paralysis?

20 specialists and care centers treating Infantile-onset ascending hereditary spastic paralysis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.