Medical Tips Online

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Raptor initiates dosing in DR Cysteamine Phase 2 clinical trial for Huntington's Disease

January 15, 2020

Huntington's Disease is a rare, progressive, and hereditary neurological disease that often leads to death within 15 to 20 years after diagnosis. The disease is thought to affect as many as 20,000-30,000 patients in the U.S. and a comparable number in Europe. There is no currently available drug that targets the defective gene believed to cause Huntington's Disease, which results in the degeneration of certain nerve cells in the brain. The disease is characterized by uncontrollable movements and...

Alnylam Pharmaceuticals reports significant progress in discovery of novel technologies for systemic delivery of RNAi therapeutics

January 14, 2020

"Progress on LNP-Mediated Delivery of RNAi Therapeutics," by Martin Maier, Ph.D., Senior Director, Drug Discovery at Alnylam; "Progress in the Development of LNP Delivery Systems for siRNA: Advancing LNPs to the Clinic," by Mark Tracy, Ph.D., Senior Director, Pharmaceutical Operations at Alnylam; "Combinatorial Development of Biomaterials and Synthetic siRNA Delivery Systems," by Professor Daniel Anderson, Ph.D., Department of Chemical Engineering, Harvard-MIT Divis...

Inhibition of MMPs can offer therapeutic benefit in Huntington's disease: Study

January 13, 2020

Dr. Ellerby, along with coauthor Dr. Robert E. Hughes and colleagues, designed a sophisticated screen to examine the generation of the smallest Htt fragments. "Our screen identified 11 proteases that, when inhibited, reduced Htt fragment accumulation," explains Dr. Hughes. "Three of these belonged to the matrix metalloproteinase (MMP) family." MMPs have been implicated in a diverse collection of pathological processes, including rheumatoid arthritis, cardiovascular disease, c...

Neuroscientist Snyder presents annual Ellis S. Grollman Lecture at UMB

January 12, 2020

Huntington's disease is caused by a mutation in the protein huntingtin (Htt) that is expressed throughout the body and all the brain regions. But the pathology of Huntington's is mostly confined to the corpus striatum, said Snyder. The Ellis Grollman Lecture in the Pharmaceutical Sciences was established at the School by his sister, Mr. Evelyn Grollman Glick, as an endowed memorial. Natalie D. Eddington, PhD, dean of the School of Pharmacy said that the annual lecture focuses on distinguished bi...

Key molecular switch linked to HD identified

January 11, 2020

The researchers found that full-length mhtt induced motor and psychiatric-like behavioral deficits, mhtt aggregation, and selective neurodegeneration in the SA mice but that these pathological changes were absent in the SD mice. In addition, SD mutations had a dramatic impact on the process of mhtt protein aggregation while SA mutations did not. Taken together, the findings demonstrate that subtle molecular changes of only two amino acids in full-length mhtt dramatically reduced the pathogenic p...