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Expert Interview

Slingshot members are talking to an expert! The topic is:

Checking in with a prescriber of Voxzogo (vosoritide) in Achondroplasia and discussing new data presented at the 2025 ACMG Meeting

Ticker(s): BMRN

Who's the expert?

Institution: Kentucky Children's Hospital 

  • Clinical geneticist and Chief, Division of Pediatric Genetics and Metabolism at Kentucky Children's Hospital
  • Manages 5 patients with achondroplasia, with 3 currently on VOXZOGO
  • Research focus includes SATB2-associated syndrome (SAS) and other genetic disorders with a particular interest in connective tissue disorders and craniofacial anomalies.

Interview Questions
Q1.

Please tell us about your clinical experience. How many patients with Achondroplasia do you see on a yearly basis? What is the standard of care, and what novel therapies are you excited about for the near future?

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Q2.

How often are any of the following procedures undertaken:-Spinal fusion to stabilize the spine- Spinal decompression to free up the compressed spinal cord or nerve roots -Guided-growth surgery/osteotomy to correct uneven growth or abnormal rotation of the bones.

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Q3.

What has been your clinical experience and feedback in terms of improved patient growth after using Voxzogo?

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Q4.

Are there other potential forms dwarfism where Voxzogo may be used as treatment?

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Q5.

What percent of your patients have been inclined to try Voxzogo? And how well do you think patients would comply with a years-long, daily, injectable schedule?

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Q6.

What are the potential risks associated with achondroplasia? How well do patients understand achondroplasia, and what percent of patients agree with treatment?

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Q7.

Baseline mean AGV in the placebo and Voxzogo groups was 4.06 cm/year and 4.26 cm/year, respectively. At week 52, the change from baseline in AGV was -0.17 cm/year for the placebo treated patients and 1.40 cm/year for the Voxzogo treated patients, resulting in a 1.57 cm/year improvement in AGV for Voxzogo patients. Could you discuss these results, how much of a positive impact would it have on the patients’ long term outcomes? How much would complications and need for surgery be reduced?

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Q8.

On a scale of 1-10, how would you rate your level of excitement for the future of this drug?

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