Researching MPS Therapies with Dr. Patricia Dickson of LA BioMed
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.
The Importance of Researcher Involvement 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.
Discussing Shire's Rare Disease Pipeline with Hartmann Wellhoefer, M.D.
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.
A Sense of Urgency in Sanfilippo Therapy Development
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.
Interim Data of Sanflippo B Treatment Presented at WORLDSymposium
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 Intends to Start Phase 3 of AGT-181 in MPS I
ArmaGen reported full 52-week results from a Phase 2 proof-of-concept study with AGT-181 in MPS I.
Are Gaucher Disease and Parkinsons Linked?
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 Presents Safety Data from Phase 1/2 in Pompe
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 Releases More Positive Data at WORLDSymposium
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.
Valerion Presents Data from Study of VAL-1221 in Pompe Disease
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.
Interim Data of Kanuma in LAL-D Infants Presented at WORLDSymposium
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.
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