Rare Metabolic Diseases - New Treatment Approaches

Rare Metabolic Diseases - New Treatment Approaches
In addition, the finding will researchers understand other protein-folding disorders, such as Parkinson's, Huntington's, and Alzheimer's diseases. Fabry disease is caused by the lack of or faulty alpha-galactosidase (α-GAL) enzyme needed to metabolize …
Read more on Medical News Today

Ultragenyx In-Licenses Therapeutic Program for Rare Genetic Disease …
While its clinical manifestations are similar to MPS 1 and MPS 2, MPS 7 is one of the rarest among the MPS disorders and is likely underdiagnosed as are most rare diseases. MPS 7 has a wide spectrum of manifestations and can present clinically as early …
Read more on MarketWatch (press release)

Targacept Inc. (TRGT) Says AstraZeneca will Advance Alzheimer's Drug
Targacept Inc. (Nasdaq: TRGT) currently has five mid-to-late-stage drug candidates using innovative NNR Therapeutics for difficult to treat diseases and disorders of the nervous system. NNR Therapeutics selectively modulates the activity of specific …
Read more on Beacon Equity Research