Overview
Achondroplasia is treated with 2 medications in our database, including VOXZOGO 0.4mg, VOXZOGO 0.56mg, VOXZOGO 1.2mg, AUKELSO. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by BioMarin, Alcon. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Achondroplasia treatment below.
Clinical phenotype terms— hover any for plain English:
Autosomal dominant
Passed on from just one parent; each child has about a 50% chance of inheriting it
Neonatal
Begins at or shortly after birth (first 4 weeks)
FDA & Trial Timeline
10 eventsSYSNAV
BioMarin Pharmaceutical — PHASE2, PHASE3
Abbisko Therapeutics Co, Ltd — PHASE1, PHASE2
QED Therapeutics, a BridgeBio company — PHASE2
Tyra Biosciences, Inc — PHASE2
Ascendis Pharma A/S — PHASE2
Kyowa Kirin Co., Ltd. — PHASE3
Ascendis Pharma Growth Disorders A/S — PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
2 availableVoxzogo 0.4Mg
VOXZOGO is indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses.
AUKELSO
* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Not Publicly Available * RxBIN, PCN, and Group numbers: Not Publi
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Financial Resources
2 resourcesYuviwel
Ascendis Pharma Growth Disorders A/S
1 travel grant are also available for Achondroplasia patients — see Travel Grants below ↓
Travel Grants
1 grantsBridgeBio Grants and Funding
Community
No community posts yet. Be the first to share your experience with Achondroplasia.
Start the conversation →Latest news about Achondroplasia
1 articlesCaregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
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.
Common questions about Achondroplasia
What is Achondroplasia?
Achondroplasia is treated with 2 medications in our database, including VOXZOGO 0.4mg, VOXZOGO 0.56mg, VOXZOGO 1.2mg, AUKELSO. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by BioMarin, Alcon. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Achondroplasia treatment below.
How is Achondroplasia inherited?
Achondroplasia follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Achondroplasia typically begin?
Typical onset of Achondroplasia is neonatal. Age of onset can vary across affected individuals.
Are there clinical trials for Achondroplasia?
Yes — 17 recruiting clinical trials are currently listed for Achondroplasia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Achondroplasia?
25 specialists and care centers treating Achondroplasia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Achondroplasia?
2 FDA-approved treatments and 3 patient support programs are currently tracked on UniteRare for Achondroplasia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.