Advocacy

From Patients to Partners: The Rising Power of Advocacy in Drug Development

David Alvaro, Ph.D.

Pharma's Almanac

Orphan Drugs

From Market Failures to Lifesaving Innovations: The Evolution of Orphan Drugs

Cynthia A. Challener, Ph.D.

Pharma's Almanac

Clinical Trials

Passion and Understanding Essential for Rare Disease Clinical Trials

Juliet Moritz

Ergomed

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

Clinical Trial Logistics

Facilitating Complex Clinical Trials For Rare Diseases

Ariette van Strien

Marken

Drug Development

Patients with Rare Leukemia Disease Will Have New Treatment Option

Nigel Walker

Nice Insight