Log In
C&EN congratulates Dan Fagin on winning the 2014 Pulitzer Prize in General Nonfiction for “Toms River: A Story of Science and Salvation” (Bantam Books). C&EN’s Rudy Baum reviewed “Toms River” last May.
Copyright © 2013 American Chemical Society
May 13, 2013 Cover
Volume 91, Issue 19
COVER: Melissa Hogan’s son Case, who has Hunter syndrome, is in an enzyme replacement clinical study. His missing enzyme is delivered directly into his cerebrospinal fluid.
Volume 91, Issue 19
After years of neglect by pharma companies, rare disease treatment is coming into the limelight
COVER: Melissa Hogan’s son Case, who has Hunter syndrome, is in an enzyme replacement clinical study. His missing enzyme is delivered directly into his cerebrospinal fluid.
Credit: Lisa Jarvis/C&EN
» Full Article
May 13, 2013 Issue
Volume 91, Issue 19
May 13, 2013 Issue, Vol. 91 | Iss. 19
After years of neglect by pharma companies, rare disease treatment is coming into the limelight
(pp. 10-12)
Features
Business
Chemists meet demand for novel ingredients that repair, add shine, and protect color (pp. 29-32)
Science & Technology
Unique properties could improve catalysis, batteries (pp. 42-43)
Cover Story: Orphans Find A Home
Orphans Find A Home
After years of neglect by pharma companies, rare disease treatment is coming into the limelight
(pp. 10-12)Small Audience, Large Payoff
The current groundswell of interest in rare diseases can be traced to Genzyme, the first firm to show that drugs for small patient populations could be profitable
(pp. 12-15)Patients Advocate For Change
Although companies ultimately bring treatments to market, it’s patient groups that are creating the awareness needed to start the drug discovery process
(pp. 15-20)Regulations Are Cause For Concern
Patient advocates and companies are encouraged by the recent introduction of new regulatory tools to speed the development of drugs for rare diseases but worry they may not go far enough for the smallest patient groups
(pp. 20-23)News of the Week
Hear, Hear For The Bionic Ear
Tissue Engineering: 3-D-printed devices detect frequencies beyond human perception
(p.5)‘Breakthrough’ Drugs Rising
Pharmaceuticals: New FDA program could cut time for approvals in half, but details are vague
(p.6)Global Ban For Flame Retardant
United Nations: Countries agree to stop use of hexabromocyclododecane under Stockholm Convention
(p.6)Accounting For Pollution
Activists in Houston are suing EPA over claims that the agency undercounts toxic air emissions
(p.7)Drug Delivery With A Bang
Nanoscience: Chain-shattering polymeric therapeutics could release medicines on demand
(p.7)A Major Award For Mass Spec
Honors: Dreyfus Prize given to R. Graham Cooks, who shrank MS devices and expanded the technique
(p.8)Robert S. Langer Receives Wolf Prize
Honors: MIT chemical engineer recognized for work in drug delivery and tissue engineering
(p.8)AstraZeneca Research Campus To Become Multicompany Bioscience Center
Bioscience: BioHub campus will host multiple drug discovery firms
(p.9)European Vinyls Giant Emerges
Plastics: Solvay, Ineos say joining together will help them tough out a rough marketplace
(p.9)Departments
Business
Financial results reflect the economic challenges of 2012, but stock values point to optimism
(pp. 26-28)
Chemists meet demand for novel ingredients that repair, add shine, and protect color
(pp. 29-32)
Patient advocates and companies are encouraged by the recent introduction of new regulatory tools to speed the development of drugs for rare diseases but worry they may not go far enough for the smallest patient groups
(pp. 20-23)
Although companies ultimately bring treatments to market, it’s patient groups that are creating the awareness needed to start the drug discovery process
(pp. 15-20)
The current groundswell of interest in rare diseases can be traced to Genzyme, the first firm to show that drugs for small patient populations could be profitable
(pp. 12-15)
After years of neglect by pharma companies, rare disease treatment is coming into the limelight
(pp. 10-12)
After years of neglect by pharma companies, rare disease treatment is coming into the limelight
(pp. 10-12)Concentrates
(pp. 24-25)
(pp. 24-25)- LG Readies Battery Plant
- Acrylonitrile Fumes Kill One In Belgium
- Kuwaiti Company Settles Up With Dow
- Chemist Not Guilty Of Secrets Theft
- Bayer Will Exit Carbon Nanotubes
- Ferro, Dissidents Reach An Agreement
- Chinese Protest Aromatics Again
- Shokubai Restarts More Plants In Japan
- ICIG Gets Another Tessenderlo Business
- Aesica Taps Academia For Amide Synthesis
- Takeda Continues Push Into Vaccines
- Ambrx And BMS Join For Drug Conjugates
- Astellas Signs Novel Development Pact
- Business Roundup
ACS News
Program set for ACS gathering in La Crosse, Wis.
(pp. 50-51)
Government & Policy
State Department program brings ACS career development training to Malaysia and Indonesia
(pp. 38-39)
Science is becoming increasingly important in diplomatic efforts at the State Department
(pp. 34-37)Education
ACS Meeting News: Courses aim to engage students who don’t know the difference between a beaker and a Bunsen burner
(pp. 46-47)
Science & Technology
Researchers obtain structure and propose mechanism of growth-signaling enzyme mTOR
(p.45)
Unique properties could improve catalysis, batteries
(pp. 42-43)Concentrates(pp. 40-41)
(pp. 40-41)- Alloy Makes Iron On The Cheap
- Airborne Mineral Dust Is Key To Cirrus Cloud Creation
- Airborne Mineral Dust Adds Sulfate To Aerosols
- Clicking Pyrroles Into Place
- Young Blood Heals Aging Hearts
- MRI Contrast Agent May Detect A Harbinger Of Cancer Drug Resistance
- Exhaust Catalyst Traps And Scrubs NOx
- Earth And Moon Share Water Source
Newscripts
Editor's Page
Most popular
Viewed
Commented
Chemistry Job Listings