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PHARMACHON • JUNE 16-18 • CHICAGO, IL

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Achondroplasia

Growing Strong

Bay Area parents Munira and Amer Haider talk about the fund they established to help support research on Achondroplasia, a form of dwarfism.

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Post Tags: #achondroplasia #funding #growing stronger #research

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Novel therapy improves height for children with achondroplasia

Key Measurement Concepts and Appropriate Clinical Outcome Assessments in Pediatric Achondroplasia Clinical Trials

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#DNADay23 – Today is the #perfectdate because to

#DNADay23 – Today is the #perfectdate because today marks 20 years since the successful completion of the Human Genome Project in 2003 and the discovery of #DNA's double helix by James Watson and Francis Crick in 1953! 🧬

Let’s continue the celebration of #genomics, specifically surrounding #achondroplasia, this summer at the 5th Annual @achondroplasiaresearch conference, @pharmachon!

This event brings together a community collaborating to understand the new innovations surrounding #FGFR3, the gene that makes up achondroplasia.

When you attend #PHARMACHON, you will have the opportunity to learn about innovations, collaborations, and advancements surrounding achondroplasia directly from the researchers and physicians themselves.

Register today through the link in bio, or by visiting pharmachon.org

#achondroplasiaawareness #achondroplasiaresearch #dwarfism #dwarfismawareness #skeletaldysplasia #growthawareness #genome #vosoritide #voxzogo #qedtherapeutics #infigratinib #ascendispharma #transconcnp #sanofi #sar442501 #tyrabiosciences #tyra300

🆕 – We are so excited to announce two new pre

🆕 – We are so excited to announce two new presentations in the conference agenda for this year!

#TyraBiosciences and @sanofi will present at the @chandlerproject's 5th Annual #AchondroplasiaResearch Conference “PHARMACHON” on June 17 in Chicago, IL.

#TYRA and #Sanofi each have a drug development pipeline for achondroplasia
• TYRA-300 ACH (Tyra)
• SAR442501 (Sanofi)

Register today to learn more about these companies and their respective pipelines for achondroplasia + new research via link in bio or by going to PHARMACHON.ORG

••••• ••••• •••••
#PHARMACHON23 #achondroplasiaawareness #achondroplasiatreatment #dwarfism #dwarfismawareness #skeletaldysplasia

DO YOU KNOW DR. RAVI…in other words, the special

DO YOU KNOW DR. RAVI…in other words, the special guest at @pharmachon??? Many of you answered and more than half of you said, “YES” 🤩

This summer, June 16-18, 2023, you can MEET Professor Ravi “Dr. Ravi” Savarirayan IN PERSON at the @chandlerproject’s 5th Annual #AchondroplasiaResearch Conference in Chicago! (#PHARMACHON) 🧬

Since it’s the fifth year of the ONLY conference in the U.S. where patients, parents & caregivers affected by #achondroplasia can hear about the latest research & developments surrounding pharmaceutical treatments (approved and in pipeline) directly from the researchers and physicians, we decided to give the conference a new name 🥳

More details about the event will be announced soon, but for now…
Registration is LIVE and Dr. Ravi CAN’T WAIT to meet you in Chicago!
June 16-18 at the @interconchicago
[ same city, weekend, and hotel as last year’s conference! ] 🏙️

EARLY BIRD REGISTRATION:
General Admission: $20 per person (children 16 and under free)
Room rate: $289/night
Early bird registration ends on Monday, April 10 @ 11:59 PM ET. After that, general admission will be $30/per person (children 16 and under free)

⭐️ LINK IN BIO TO REGISTER ⭐️

Note: This year’s conference is exclusively in person. There will not be a virtual attendance option.

Follow @pharmachon for all information and up-to-date details about the event

#PHARMACHON23 #achondroplasiaawareness #achondroplasiatreatment #dwarfism #dwarfismawareness #skeletaldysplasia #childhealth #MurdochChildrens #BioMarin #voxzogo #vosoritide #Pfizer #BridgeBio #QEDtherapeutics ##QEDtx #Infigratinib #AscendisPharma #TransCon #TransConCNP #TyraBioSciences

Do you know a student with #achondroplasia who is

Do you know a student with #achondroplasia who is preparing to apply to college? If so, let them know about @biomarinofficial’s RARE Scholars Program! 🎓

#RAREScholars is an annual scholarship opportunity for students living with a #genetic condition. These scholarships recognize and support students who have demonstrated exceptional leadership and engagement in school and community activities.

Students who apply have a chance to receive up to $5000 in scholarship funds. The deadline to apply is 𝗪𝗘𝗗𝗡𝗘𝗦𝗗𝗔𝗬, 𝗠𝗔𝗥𝗖𝗛 𝟮𝟵 𝗮𝘁 𝟯 𝗣.𝗠. 𝗖𝗘𝗡𝗧𝗥𝗔𝗟 𝗧𝗜𝗠𝗘 (next week!)

For more information about the scholarship, eligibility to apply, and the application, 𝙘𝙡𝙞𝙘𝙠 𝙩𝙝𝙚 𝙡𝙞𝙣𝙠 𝙞𝙣 𝙗𝙞𝙤.

#achondroplasiaresearch #achondroplasiaawareness #FGFR3 #dwarfism #dwarfismawareness #skeletaldysplasia #BioMarin #academic #education #scholarship #graduate #graduation #classof2023 #college #collegebound

NEW! MARCH 7, 2023 – FDA Accepts #BioMarin's Sup

NEW! MARCH 7, 2023 – FDA Accepts #BioMarin's Supplemental New Drug Application to Expand Use of #VOXZOGO® (#vosoritide) for Injection to Treat Children with #Achondroplasia Under the Age of 5

The Supplemental New Drug Application is Based on Positive Results from Global Randomized Phase 2 Study in Infants and Young Children

FDA set PDUFA Target Action Date of October 21, 2023

LINK IN BIO FOR FULL PRESS RELEASE

#achondroplasiaresearch #achondroplasiaawareness #achondroplasiatreatment #FGFR3 #dwarfism #dwarfismawareness #skeletaldysplasia #biopharma #bionews #biotech #biotechnology #pharmaceutical

#REPOST from @orthodoctorphil, Assistant Director

#REPOST from @orthodoctorphil, Assistant Director of @limblengthmd at @lifebridgehealth –

"Achondroplasia is the most common skeletal dysplasia. Fortunately, new medications have allowed medical treatment for aspects of Achondroplasia that were previously untreatable or required surgical correction.

Will the medical interventions be enough to restore functional height (>143cm) and humeral length to allow smooth activities of daily living?

Only time will tell...

For patients who have missed the window for medical intervention, or who are looking for additional treatment, distraction osteogenesis provides excellent results under expert supervision.

In this patient over 10cm of height were gained and over 10cm of length gained in the humerus, improving function.

#pediatricorthopedics #skeletaldysplasia #pediatricorthopaedics #limblengthening #limblenghteningsurgery #distraction #ilizarov #precice #externalfixator @limblengthmd @bonelengthening @chandlerproject”
___
#achondroplasia #achondroplasiaresearch #achondroplasiaawareness #hypochondroplasia #pseudoachondroplasia #diastrophicdysplasia #dwarfism #dwarfismawareness

MARCH 6, 2023 – #BridgeBio Announces Positive Ph

MARCH 6, 2023 – #BridgeBio Announces Positive Phase 2 Cohort 5 Results of #Infigratinib in #Achondroplasia Demonstrating Mean Increase in Annualized Height Velocity of 3.03 cm/year with No Treatment-related Adverse Events

– In the highest dose level (Cohort 5, 0.25 mg/kg once daily), the mean change from baseline in annualized height velocity (AHV) at six months was +3.03 cm/yr (p = 0.0022) for the first 10 children with at least six months of follow-up in Cohort 5. The two remaining children who have not yet had six months of follow-up have a mean change from baseline in AHV of +8.8 cm/yr based on three months data

– 80% of children at six months were responders, as defined by an increase from baseline AHV of at least 25%. The mean change from baseline in AHV of responders was 3.81 cm/yr

– As a result of treatment, the median absolute AHV reached 7.6 cm/yr, which is beyond the 99th percentile of growth for children living with achondroplasia

– Infigratinib demonstrated clear dose-responsiveness as a single daily oral therapy and was well-tolerated with no adverse events (AEs) assessed as treatment-related in Cohort 5

– Based on the positive Phase 2 results, BridgeBio has started to enroll children for a pivotal Phase 3 trial

– BridgeBio expects to initiate clinical development of infigratinib for #hypochondroplasia, a skeletal dysplasia closely related to achondroplasia and driven by fibroblast growth factor receptor 3 (FGFR3) gain-of-function variants, and will continue to explore the impact on the medical and functional complications of achondroplasia in future studies of infigratinib

LINK IN BIO FOR FULL PRESS RELEASE

#achondroplasiaresearch #achondroplasiatreatment #achondroplasiaawareness #hypochondroplasiaresearch #hypochondroplasiaawareness #dwarfism #dwarfismawareness #skeletaldysplasia #biopharma #bionews #biotech #biotechnology #pharmaceutical #QEDtx #QEDtherapeutics

The #BridgeBio management team will be sharing the

The #BridgeBio management team will be sharing their Phase 2 data results for #infigratinib in #Achondroplasia on Monday, March 6th at 7:30 am ET.

Register for the webinar via link in bio

#achondroplasiaresearch #achondroplasiatreatment #achondroplasiaawareness #dwarfism #dwarfismawareness #skeletaldysplasia #biopharma #bionews #biotech #biotechnology #pharmaceutical #QEDtx #QEDtherapeutics

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