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Therefore, the applications of such molecules can enhance the functional deficiency of enzymes in lysosomal storage diseases (whose genes are not extensively damaged, and thus correctable with folding therapy).
"Affecting one in 35,000 newborns worldwide, Fabry disease is a rare lysosomal storage disorder due to a key enzyme deficiency.
Our analytical research validates the bearish argument for Amicus in the short term yet supports the bullish case in the long haul. In the second half of 2015, the stock enjoyed substantial gains, followed by the tumultuous tumbling in share price due to the FDA rejection of its lead molecule, migalastat (Galafold).
Based in Cranbury, New Jersey, Amicus Therapeutics (NASDAQ: FOLD), a biotechnology at the forefront of individualized medicine, is focusing on the development of a novel chaperone therapeutic - that either stabilizes a defective protein by itself or in combination with existing enzyme replacement therapies ("ERTs").
Poor information exchange often includes inaccurate or missing information regarding patients current health status, medication plan, functional and psychological history and unresolved plans for follow-up care post discharge or transfer from a hospital to another facility.4 7 Patients and their caregivers (eg, family) may then be uninformed or misinformed about their illness and medications and not be able to carry out the care transitions plan or manage their own health.9 The exchange and transfer of accurate and complete information to patients and their caregivers around what is currently happening and what to expect next is an important component of ensuring quality care transitions.8 10 11 By providing information to patients (and when available their caregivers), they are more informed and have the potential to actively participate in their care8 which is associated with improved patient satisfaction and clinical outcomes.12 However, varying degrees of information exchange with patients13 and their subsequent participation in their care planning11 14 have been reported.
Thereafter, the share price climbed steadily and rewarded investors with 55% appreciation.
Objective To explore the perceptions of patients, their caregivers and healthcare professionals associated with the exchange of information during transitioning from two acute care hospitals to one rehabilitation hospital.
Participants and setting Patients over the age of 65 years admitted to an orthopaedic unit for a non-elective admission, their caregivers and healthcare professionals involved in their care.
Upper panel: Snapshot showing extensive cluster formation observed in lower order polysulfides and decreases in the tendency to form such polymeric structures with addition of lithium salt.
Lower panel: Radial distribution function of (a) Li-Li in Li2Sx (x= 2 to 8) and Li-TFSI in Li2Sx (x= 2 to 8) Li(TFSI) in DOL: DME solvent A coupled theoretical and experimental study of bulk solvation structure and transport properties of lithium salt (Li-TFSI) and polysulfides species revealed extensive cluster formation in lower order polysulfides (S4) show high solubility and slow dynamics in the solution.
Participating sites included orthopaedic inpatient units from two acute care teaching hospitals and one orthopaedic unit at a rehabilitation hospital in an urban setting.