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FDA approves AMVUTTRA for treatment of hATTR amyloidosis with polyneuropathy
BREAKING NEWS: The U.S. Food and Drug Administration today approved Alnylam Pharmaceuticals AMVUTTRATM, a novel therapy for the treatment of hereditary transthyretin (hATTR) amyloidosis with polyneuropathy. This approval comes after positive results from the readout of HELIOS-A Phase III study in which patients on the RNAi therapeutic met all secondary endpoints measured at 18 months. “Hereditary ATTR amyloidosis is an aggressive, debilitating, and often fatal disease,” says Michael Polydefkis, Director of the Cutaneous Nerve Lab and Professor of Neurology at...
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Neuropathy in Amyloidosis
Many types of amyloidosis can affect patients’ nerves, causing painful, annoying, or even debilitating symptoms. It is common for patients with amyloidosis to experience neuropathy and in fact, some patients may have nerve-related symptoms as their most predominant sign or chief complaint.
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Results From The ATTR Affordability Study
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Amyloidosis Forum highlighted as an exemplary model to enhance product development
The Amyloidosis Forum, a public-private partnership initiated between the Amyloidosis Research Consortium and FDA, was highlighted recently during FDA Rare Disease Day Virtual Public Meeting on March 4th as an exemplary model to enhance product development using a public-private partnership approach. Launched in 2019, the Amyloidosis Forum brings together the entire amyloidosis community- from patients to academia, regulators, industry, and other related research fields to, partner on key initiatives that are designed to bridge gaps in regulatory science and ultimately...
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Amyloidosis Community Survey
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