Alan Beggs, PhD, on Challenges in Therapeutic Development for Rare Diseases
The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed takeaways from an MDA session on rare diseases.
Alan Beggs, PhD, on Lessons Learned With Developing X-Linked Myotubular Myopathy Gene Therapy
The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed takeaways from a session on rare disease gene therapy at MDA.