Achondroplasia Insights

This week's must-know community updates, latest research & events

April 02, 2025 • Estimated Reading Time: 1 minute

Top Stories

Navepegritide NDA submitted for achondroplasia treatment
Navepegritide, given once a week, aims to increase growth in children with achondroplasia by continuously exposing growth plates to a growth-promoting peptide. The treatment has shown improved growth rates and muscle function in clinical trials.
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FDA Approves Diazoxide Choline for Prader-Willi Syndrome
While this approval doesn't directly affect achondroplasia, it shows progress in treating genetic conditions. Diazoxide choline helps manage excessive hunger in Prader-Willi syndrome, a condition distinct from achondroplasia but also impacting growth and development.
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FDA Approves New Treatments for Children
New treatments for various conditions were approved, including a submission for achondroplasia treatment, which could potentially improve growth in affected children.
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Latest Research

No latest research available.

Clinical Trials

This is a list of upcoming or ongoing clinical trials that are actively recruiting and have been listed or updated in the last two weeks:

Community News

Dwarf Athletic Association of AmericaDwarf Athletic Association of AmericaApr 02, 2025

Thumbs up if you were so excited about going to San Diego that you crashed the LPA conference registration system today!

The Chandler ProjectThe Chandler ProjectApr 01, 2025

#AscendisPharma A/S Submits U.S. NDA for #TransConCNP (#Navepegritide) for the Treatment of Children with #Achondroplasia

🔹 Data demonstrated multiple clinical benefits beyond linear growth

🔹 NDA supported by data from three randomized, double-blind, placebo-controlled clinical trials in children with achondroplasia, with up to three years of open-label extension data

🔹 MAA in EU on track for submission during Q3 2025

The Chandler Project Post
The MAGIC FoundationThe MAGIC FoundationMar 29, 2025

Need help covering the cost of attending The MAGIC Foundation's 31st Annual Convention? Apply for a MAGIC Foundation scholarship and join us for this life-changing event!

📅 Convention Dates: July 10-13, 2025

📍 Location: Lombard, IL

🔗 Apply through the link in our program: https://www.magicfoundation.org/31st-annual-convention

The MAGIC Foundation Post

Upcoming Events

APR
13
5K Run & Walk for Kids' GrowthIn Person Human Growth Foundation • Greenvale, NY
JUL
03
LPA National ConferenceIn Person Little People of America • Sonoma, CA
OCT
24
Annual ConventionIn Person Restricted Growth Association • Presteigne, United Kingdom