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Rare Disease Day 2020: Progress toward cures
By Richard Moscicki, M.D. | February 24, 2020
Today kicks off Rare Disease Week, an annual opportunity to celebrate the incredible progress that has been made to treat rare diseases and reflect on the need for continued research and...
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Speaker Pelosi’s drug pricing plan could result in 56 fewer new medicines over 10 years
By Juliet Johnson | November 21, 2019
A new analysis from economic consulting firm Vital Transformation shows that Speaker Nancy Pelosi’s drug pricing plan, H.R.3, could result in at least 56 fewer innovative medicines for patients...
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Pelosi’s radical plan would leave patients with sickle cell disease behind
By Tom Wilbur | November 14, 2019
Sickle cell disease (SCD) is the most commonly inherited genetic blood disorder, affecting nearly 100,000 children and adults in the United States. Three million Americans carry the sickle cell...
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Pelosi’s radical plan would leave patients with rare pediatric diseases behind
By Tom Wilbur | November 7, 2019
There are about 7,000 rare diseases and half of these affect children. According to the National Institutes of Health (NIH), 30 million Americans, or 10 percent of the population, have one of the...
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ICYMI: Fighting for a cure to ALS
By Anne McDonald Pritchett, PhD | September 25, 2019
Yesterday at The Atlantic Festival, I had the chance to speak with Amy, a biopharmaceutical researcher focused on amyotrophic lateral sclerosis (ALS). As a young girl, Amy’s father inspired her to...
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New report shows nearly 20 innovative medicines in development for sickle cell disease
By Andrew Powaleny | April 2, 2019
Sickle cell disease (SCD) is the most common inherited blood disorder in the United States, afflicting nearly 100,000 Americans. Patients with SCD have sickle-shaped red blood cells, which have...
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Guest post: Hearing from patients on Rare Disease Day
By Guest Contributor | February 28, 2019
Conversations and healthy debate about issues facing our industry and the health care system are critical to addressing some of today’s challenges and opportunities. The Catalyst welcomes guest...
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Rare Disease Day: Unmet medical need inspires biopharmaceutical innovation
By Richard Moscicki, M.D. | February 28, 2019
Today is Rare Disease Day, an annual reminder of the incredible community of patients living with a rare disease. In the U.S., rare diseases are classified as conditions that affect fewer than...
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Putting 2018 medicine approvals in context
By Andrew Powaleny | January 23, 2019
Last year, the U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved a record number of novel new medicines with 59 new molecular entities (NMEs). While...
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ICYMI: Orphan drug development brings unique challenges
By Gretta Stone | May 21, 2018
A new report from the Tufts Center for the Study of Drug Development finds that it takes 2.3 years, or 18 percent, longer to develop an orphan drug compared with medicines to treat more common...
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