Expert Interview
Examining the current treatment landscape and anti myostatin inhibition approaches for Spinal Muscular Atrophy (SMA) including Scholar Rock's apitegromab, Biohaven's taldefgrobep Alfa and Roche's GYM329
Ticker(s): SRRK, RHHBYInstitution: Connecticut Children’s
- Pediatric neurologist and Division Head of Neurology at Connecticut Children’s
- Clinical and research interests include spinal muscular atrophy, muscular dystrophies, Charcot-Marie-Tooth disease, and genetic treatments for neurological conditions.
- Manages 27 patients with SMA and can speak to the anti myostatin inhibition approaches for SMA including Scholar Rock's apitegromab, Biohaven's taldefgrobep Alfa and Roche's GYM329
How many patients do you manage with SMA?
Added By: dami_adminOn a scale from 1-10 (10 being extremely excited) where would you rate your level of excitement for apitegromab
Added By: dami_admin- What is the current treatment landscape for Spinal Muscular Atrophy (SMA)?
- What are the anti-myostatin inhibition approaches for SMA?
- Can you give some background on the type of patients you treat and the number of patients in your care?
- What treatments do you utilize for spinal muscular atrophy?
- What is the breakdown of ambulatory versus non-ambulatory patients in your care?
- How is the landscape shifting in terms of patient mobility given the gene therapy?
- How do you make treatment decisions between Evrysdi, Spinraza, and Zolgensma?
- What is the breakdown between Evrysdi and Spinraza for later onset patients?
- Why would a patient choose intrathecal injection over oral administration?
- Do you think patient preferences will change going forward?
- What are your thoughts on the higher dose trial for Spinraza?
- Do you think there is a scientific backing for a higher dose of Spinraza?
- What is the situation with therapies after Zolgensma?
- How would you describe the current unmet need for SMA patients?
- What specific patient populations do you think would benefit from myostatin targeting therapies?
- Do you think there is an advantage to targeting active myostatin versus latent myostatin?
- What are the logistical differences between IV and subcutaneous administration?
- Assuming efficacy is the same, is it an easy switch from IV to Sub-Q for myostatin therapies?
- What are your thoughts on the trial results from Scholar Rock?
- How clinically meaningful is a 4.4 change on the HFMSE scale?
- Do the long-term results of nusinersen and risdiplam agree with your clinical experience?
- Have you seen any differences in long-term efficacy between risdiplam and nusinersen?
- Is there a difference in the plateauing effect for patients less than five years old versus older?
- Do you think there is potential utility in treating patients after Zolgensma with myostatin therapy?
- What percentage of your patients do you think would be getting Evrysdi versus Spinraza in two years?
- How important is physical exercise in influencing the results of these treatments?
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- Apitegromab Phase 3 sapphire results SRRK Future Window: Oct 01, 2024 - Dec 31, 2024
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