Clinical Trials

Optimizing Oncology and Rare Disease Clinical Trials by Focusing on Science and Aligning with both Patients and Investigators

Dave Selkirk

Ergomed

Rare Diseases

Staying Agile to Bring Therapies to Patients with Rare Diseases

Sheila Frame

Amryt Pharma

Rare Disease Clinical Trials

Rare Disease Patients Benefit from Clinical Trial Flexibility

Tim Rich; Meredith McCumbee; Francis Jones, Ph.D.

PPD, part of Thermo Fisher Scientific

Rare Diseases

Leveraging AI and RNA to Fight Rare Diseases

Envisagenics

Gene Therapy

Beyond 2020: Looking into the Crystal Ball for Gene Therapy

Ratish Krishnan

MilliporeSigma

Endocrine Diseases

Developing Transformative Treatments for Rare Endocrine Diseases

Abraham N. Ceesay

Tiburio Therapeutics

Drug Approval

U.S. FDA Approves VYNDAQEL® and VYNDAMAX™ for Use in Patients with Transthyretin Amyloid Cardiomyopathy, a Rare and Fatal Disease

Pfizer Rare Disease

Clinical Trial Logistics

Facilitating Complex Clinical Trials For Rare Diseases

Ariette van Strien

Marken

Cell and Gene Therapy

REGENXBIO and Abeona Therapeutics Announce Worldwide Exclusive Licenses for the Treatment of Four Rare Lysosomal Storage Disorders Using NAV AAV9 Vector

REGENXBIO

Drug Development

Patients with Rare Leukemia Disease Will Have New Treatment Option

Nigel Walker

Nice Insight

Business

Expansion Therapeutics Raises $55.3 Million in Series A Financing to Advance Portfolio of Novel RNA Targeted Small Molecule Medicines to Treat Rare Diseases

Expansion Therapeutics, Inc.