Held April 16 and 17, this FDA workshop focused on cognitive endpoints in rare inborn errors of metabolism and common diseases. These presentations (video, transcripts, and slides) are now on-line.
UK Mucopolysaccharidosis Society Workshop
The UK Mucopolysaccharidosis Society had an important recent workshop regarding natural history studies and clinical trials in Sanfilippo syndrome (Mucopolysaccharidosis Type III) that included academia, industry, and advocacy groups. This workshop concluded that common natural history databases and standard protocols were necessary to ensure that patients obtain the best services.