Amicus Therapeutics Inc.'s shares rocketed 20.4 percent Wednesday as the company delivered positive top-line data from a second phase III study of migalastat, its oral small-molecule chaperone for Fabry disease patients. more »
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- 'Wellcome' effort to speed development of Ebola drugs, vaccines
- Injected vaccine is shot in the arm for polio eradication
- Raise the 'Curtana' for start-up with $7.6M gran...
At the beginning of this month the FDA released its much anticipated final guidance for companion diagnostics tests. It recognizes that those tests are evolving as essential components in order for new innovative therapies to be targeted to patients who will benefit the most.
Scientists from Roche Pharma Research and Early Development (pRED) and PTC Therapeutics Inc., with colleagues in academia, reported on small-molecule drugs that improved the symptoms of spinal muscular atrophy (SMA) by altering the splicing of the protein SMN2.
In mouse models of SMA, the compounds "profoundly altered the course of the disease," PTC Therapeutics CEO and co-author Stuart Peltz told BioWorld Today.READ MORE »
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- Experimental drug fights Ebola's cousin Marburg, even after symptom onset »
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- Send in the 'caveolaery' and solve drug delivery problems »
- Part biosurgery, part immunotherapy, C. novyi destroys tumor »
- Researchers resolve bone development debate »
- Best way to look at inflammation data is in dispute »
- Stem cell survival factor could improve bone marrow transplants »
- A little irony here? FDA’s technical difficulties on social media webinar send folks a’Twitter’July 10, 2014 | 9:44 PM | Posted by: Marie Powers
- Old news, yet still making headlinesJune 13, 2014 | 4:11 PM | Posted by: Mari Serebrov
- Pigoons, politicians and the periodic table: BioWorld’s 8th annual biotech summer reading listJune 06, 2014 | 4:09 PM | Posted by: Marie Powers
- ASCO strides provide cold comfortJune 05, 2014 | 4:38 PM | Posted by: Randy Osborne
- Capturing cancer drugs' true value calls for clear thinkingMay 30, 2014 | 3:48 PM | Posted by: Michael Fitzhugh
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