Tuesday, May 5, 2015

U.S. clinics avoiding government oversight of "stem cell" treatments

Federal Regulatory Oversight of US Clinics Marketing Adipose-Derived Autologous Stem Cell Interventions: Insights From 3 New FDA Draft Guidance Documents. Leigh G. Turner, PhD, Mayo Clinic Proceedings, Volume 90, Issue 5, May 2015, Pages 567–571

Chondrial Therapeutics is pleased to announce several development milestones. Update 05.15

Chondrial Therapeutics is pleased to announce several development milestones. Update 05.15.

May 1, 2015

Chondrial Therapeutics is pleased to announce several development milestones. The company has successfully manufactured its lead commercial drug candidate for Friedreich’s Ataxia. It has also initiated preclinical validation studies and preliminary results are very encouraging. The company plans on pre-IND discussions later this year and conducting GLP Toxicology studies early next year.

PMPCA mutations cause abnormal mitochondrial protein processing in patients with non-progressive cerebellar ataxia

PMPCA mutations cause abnormal mitochondrial protein processing in patients with non-progressive cerebellar ataxia. Rebekah K. Jobling , Mirna Assoum , Oleksandr Gakh , Susan Blaser , Julian A. Raiman , Cyril Mignot , Emmanuel Roze , Alexandra Dürr , Alexis Brice , Nicolas Lévy , Chitra Prasad , Tara Paton , Andrew D. Paterson , Nicole M. Roslin , Christian R. Marshall , Jean-Pierre Desvignes , Nathalie Roëckel-Trevisiol , Stephen W. Scherer , Guy A. Rouleau , André Mégarbané , Grazia Isaya , Valérie Delague , Grace Yoon; Brain. 2015 Mar 25. pii: awv057. [Epub ahead of print] DOI: http://dx.doi.org/10.1093/brain/awv057

In particular, this mutation impacts the maturation process of frataxin, the protein which is depleted in Friedreich ataxia.