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Data Roundup: March 2025 Features Updates from MDA's Conference, an Alzheimer Disease Trial, and More
Data Roundup: March 2025 Features Updates from MDA's Conference, an Alzheimer Disease Trial, and More

April 1st 2025

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

FDA Activity Recap: March 2025 Features FDA Approvals in Hemophilia, ATTR Amyloidosis, and More
FDA Activity Recap: March 2025 Features FDA Approvals in Hemophilia, ATTR Amyloidosis, and More

April 1st 2025

Robert Califf, MD, MACC, a cardiologist and former FDA commissioner
Urgency of Funding and Equity in Neuromuscular Research and Care

March 31st 2025

Natalie Goedeker, CPNP, the codirector of pediatric neuromuscular clinical research at Washington University in St. Louis
Handling Neuromuscular Gene Therapy at Real-World Sites

March 30th 2025

Video Interviews

Conference Coverage

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Natalie Goedeker, CPNP, on Handling Neuromuscular Gene Therapy at Real-World Sites
Natalie Goedeker, CPNP, on Handling Neuromuscular Gene Therapy at Real-World Sites

March 27th 2025

Sarepta’s DMD Gene Therapy Elevidys Demonstrates Manageable Safety Across 5-Year Timespan
Sarepta’s DMD Gene Therapy Elevidys Demonstrates Manageable Safety Across 5-Year Timespan

March 26th 2025

REGENXBIO’s DMD Gene Therapy RGX-202 Generates Microdystrophin Expression in Additional Patients Treated in Phase 1/2 Trial
REGENXBIO’s DMD Gene Therapy RGX-202 Generates Microdystrophin Expression in Additional Patients Treated in Phase 1/2 Trial

March 20th 2025

Genethon’s Gene Therapy GNT0004 Continues to Show Potential in DMD
Genethon’s Gene Therapy GNT0004 Continues to Show Potential in DMD

March 20th 2025

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