C&EN Senior Editor Lisa Jarvis notes that until recent interest from big pharmaceutical companies, a small drug company in Cambridge, Mass. named Seaside Therapeutics was virtually the only company trying to develop drugs for autism and fragile X syndrome.
Diagnoses of autism and related conditions termed autism spectrum disorders have increased dramatically since the 1980s, for reasons not yet fully clear. They affect millions of people worldwide. Fragile X syndrome, the most common known genetic cause of autism, results from mutation in a single gene. Its symptoms range from learning impairment to mental retardation. The disease affects about 1 in 4,000 males and 1 in 6,000 to 8,000 females.
The article describes how Seaside, armed with funding from an anonymous wealthy family and new insights into the basic science behind these disorders, is making progress toward treating these much-neglected diseases. Two of the company's potential drugs show promise in clinical trials as treatments for Fragile X syndrome. One appears to improve the behavior of children with severe social impairments. On the heels of Seaside's encouraging results, big pharmaceutical companies that once showed little interest in tackling these diseases are now trying to develop their own new medications.
ARTICLE FOR IMMEDIATE RELEASE "Tackling Fragile X"
This story is available at http://pubs.acs.org/cen/business/88/8837bus3.html
Michael Bernstein | EurekAlert!
Ion treatments for cardiac arrhythmia — Non-invasive alternative to catheter-based surgery
20.01.2017 | GSI Helmholtzzentrum für Schwerionenforschung GmbH
Seeking structure with metagenome sequences
20.01.2017 | DOE/Joint Genome Institute
An important step towards a completely new experimental access to quantum physics has been made at University of Konstanz. The team of scientists headed by...
Yersiniae cause severe intestinal infections. Studies using Yersinia pseudotuberculosis as a model organism aim to elucidate the infection mechanisms of these...
Researchers from the University of Hamburg in Germany, in collaboration with colleagues from the University of Aarhus in Denmark, have synthesized a new superconducting material by growing a few layers of an antiferromagnetic transition-metal chalcogenide on a bismuth-based topological insulator, both being non-superconducting materials.
While superconductivity and magnetism are generally believed to be mutually exclusive, surprisingly, in this new material, superconducting correlations...
Laser-driving of semimetals allows creating novel quasiparticle states within condensed matter systems and switching between different states on ultrafast time scales
Studying properties of fundamental particles in condensed matter systems is a promising approach to quantum field theory. Quasiparticles offer the opportunity...
Among the general public, solar thermal energy is currently associated with dark blue, rectangular collectors on building roofs. Technologies are needed for aesthetically high quality architecture which offer the architect more room for manoeuvre when it comes to low- and plus-energy buildings. With the “ArKol” project, researchers at Fraunhofer ISE together with partners are currently developing two façade collectors for solar thermal energy generation, which permit a high degree of design flexibility: a strip collector for opaque façade sections and a solar thermal blind for transparent sections. The current state of the two developments will be presented at the BAU 2017 trade fair.
As part of the “ArKol – development of architecturally highly integrated façade collectors with heat pipes” project, Fraunhofer ISE together with its partners...
19.01.2017 | Event News
10.01.2017 | Event News
09.01.2017 | Event News
20.01.2017 | Awards Funding
20.01.2017 | Materials Sciences
20.01.2017 | Life Sciences