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The U.S. Food and Drug Administration released new draft guidance that addresses Breakthrough Therapy designation.

On Tuesday morning, Eledon Pharmaceuticals announced positive topline results from the company’s Phase IIa clinical trial evaluating tegoprubart in patients with amyotrophic lateral sclerosis (ALS).

Diversity in all clinical research—including rare disease research—is currently under scrutiny, partly because of disparities experienced with COVID-19. Rightly so, as data show that 86 percent of genomics studies to date have involved people of European ancestry, which has limited the potential benefits of genomic research for many populations. Rebecca Sutphen, Co-Founder and Chief Medical Officer of InformedDNA, and Board-Certified Genetic Counselor Karmen Trzupek analyze strategies to increase diversity in rare genetic disease trials.

Eisai Co. Ltd. initiated Phase 3 clinical trials of the Alzheimer’s treatment BAN2401 one day after the Japanese drugmaker and U.S. partner Biogen Inc. scrapped studies for the Alzheimer’s drug aducanumab.

There was a significant drop-off in NME approvals by FDA in 2016, but there were not enough new drug applications with user fee goals to reach the lofty 2015 total.

WASHINGTON, July 19, 2015 /PRNewswire-USNewswire/ — Promising early results of new drugs that target common components of several brain diseases that cause dementia – including Alzheimer’s disease, Parkinson’s disease, and Lewy Body dementia – were reported today at the Alzheimer’s Association International Conference® 2015 (AAIC® 2015) in Washington, D.C. These diseases cause a range of […]