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Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder of upper and lower motor neurons that results in progressive motor impairment. ALS is the most common disease of motor neurons with an annual incidence of approximately 1.7–2.5 per 100,000 people. It is a terminal condition with a typical life expectancy of 2–5 years from symptom onset. […]

Tahseen Mozaffar: PROPEL Study Results Investigating the use of AT-GAA for the Treatment of Late-onset Pompe Disease

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Published Online: Mar 15th 2021

touchNEUROLOGY caught up with Tahseen Mozaffar (University California, Irvine, CA, USA) to discuss the results from the PROPEL study (NCT03729362) investigating the use of AT-GAA for the treatment of late-onset Pompe disease.

Questions:

  1. Could you tell us a little about late onset Pompe disease and the unmet needs in its treatment? (0:15)
  2. What is AT-GAA and what is its mechanism of action? (1:28)
  3. What clinical evidence to date supports the use of AT-GAA for the treatment of late-onset Pompe disease? (3:29)
  4. What are the aims and design of the PROPEL study? (7:51)
  5. What are the clinical endpoints of the study? (8:44)

Disclosures: Tahseen Mozaffar has served as an advisor to Amicus on trial design; and has served as an investigator on Amicus’ clinical trials on Pompe Disease.

Support: Interview and filming supported by Touch Medical Media

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