WORLDSymposium

Patricia Dickson, M.D., discusses similarities between variations of MPS and the both the benefits and challenges of being a researcher in the rare disease community.

Alexy V. Pshezhetsky, Ph.D., professor at the University of Montreal. sits down at WORLDSymposium to stress the importance of researcher involvement throughout the rare disease community.

Hartmann Wellhoefer, M.D., head of medical affairs at Shire, sits down with RDR at the 14th Annual WORLDSymposium to discuss the company's recent data for lysosomal storage diseases.

With no real standard-of-care for Sanfilippo syndrome patients, Brian Bigger, Ph.D., discusses the sense of urgency he and his colleagues feel while researching.

BioMarin presented interim data at the 14th Annual WORLDSymposium in San Diego from a Phase 1/2 trial for BMN 250 in MPS IIIB.

ArmaGen reported full 52-week results from a Phase 2 proof-of-concept study with AGT-181 in MPS I.

Rare Disease Report sits down with Simon Heales, Ph.D., Professor of Clinical Chemistry at Great Ormond Street Children’s Hospital in London who discusses a potential link between Gaucher disease and Parkinson’s disease.

Sanofi Genzyme presented results from the Phase 1/2 trial, showing consistent safety data for the investigational avalglucosidase alfa in late-onset Pompe disease.

Amicus presented additional positive results from the company’s Phase 1/2 clinical study of ATB200/AT2221 in patients with Pompe disease in a late-breaker poster and a corresponding oral presentation.

Today at the WORLDSymposium in San Diego, Valerion Therapeutics presented initial results from the first cohort of its ongoing Phase 1/2 clinical study of VAL-1221 in patients with late-onset Pompe disease.

At the 14th Annual WORLDSymposium, interim data from two open-label studies were presented, exhibiting a 3-year survival estimate of 68% in infants with rapidly progressing LAL-D treated with sebelipase alfa.