Videos

The professor from National Taiwan University Hospital discussed his work in improving diagnoses in Pompe disease and AADC deficiency.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the importance of accessibility in clinical trials.

The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed demographics of the HOPE-B study.

The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed research with AAV gene therapy in hemophilia.

The global head of research and development at Atara Biotherapeutics discussed the future of MS research.

The associate professor from UC San Diego discussed the ongoing phase ½ trial of CTNS-RD

The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed potential effects of cilta-cel's approval.

The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.

The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.

The chief executive officer and director of Freeline Therapeutics discussed the importance of Rare Disease Day.

Jennifer Buell, PhD, president and chief executive officer, MiNK Therapeutics, discussed the company’s iNKT cell platform.

The chief scientific officer of the CMT Research Foundation discussed the foundation’s mission.

David Taylor, PhD, an assistant professor in the department of molecular biosciences at UT Austin, discussed the Cas9 research conducted between 2 labs at UT Austin.

The senior vice president and head of clinical development at Mustang Bio discussed the importance of Rare Disease Day.

The oncologists from MD Anderson and Memorial Sloan Kettering Cancer Centers discuss sequencing CAR T-cell therapies and other key therapies in patients with B-cell acute lymphoblastic leukemia.

The chief scientific officer of FibroBiologics discussed efficacy findings from a phase 1/2 trial.

The cofounder and chief scientific officer of Cartesian Therapeutics discussed the importance of Rare Disease Day.

The professor from National Taiwan University Hospital discussed the importance of Rare Disease Day for raising awareness.

The president and chief operating officer of Rocket Pharmaceuticals discussed the importance of Rare Disease Day.

The director of the Powell Gene Therapy Center at the University of Florida discussed the importance of Rare Disease Day.

The professor of ophthalmology from Radboud University, The Netherlands, discussed ongoing gene therapy trials in Stargardt disease.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the organization’s initiative to advance genetic testing.

The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed mitigation strategies in trials and clinic.

The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed the advantages of AAV gene therapies.

The chief scientific officer of FibroBiologics discussed the benefits of fibroblast cell technology in MS.

The ophthalmologist from John A. Moran Eye Center discussed the close association between disease progression and uncovered risk alleles.

Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed FCX-013 for the potential treatment of scleroderma.

The global head of research and development at Atara Biotherapeutics discussed the link between EBV and MS.