Concerns over Efficacy and Cost of Muscle Wasting Treatments
Concerns over Efficacy and Cost of Muscle Wasting Treatments
Two new medications for treating a rare and deadly neuromuscular disease have high prices and questionable efficacies, say scientists.
Concerns over Efficacy and Cost of Muscle Wasting Treatments
Concerns over Efficacy and Cost of Muscle Wasting Treatments

Two new medications for treating a rare and deadly neuromuscular disease have high prices and questionable efficacies, say scientists.

Two new medications for treating a rare and deadly neuromuscular disease have high prices and questionable efficacies, say scientists.

inherited disease
Recent Trials for Fragile X Syndrome Offer Hope
Recent Trials for Fragile X Syndrome Offer Hope
Randi Hagerman | Sep 1, 2019
Despite a solid understanding of the biological basis of fragile X syndrome, researchers have struggled to develop effective treatments.
Infographic: The Genetics of Fragile X Syndrome
Infographic: The Genetics of Fragile X Syndrome
Randi Hagerman | Sep 1, 2019
Variation in the number of CGG repeats in the FMR1 gene at the bottom of the X chromosome can lead to increased levels of mRNA or decreased levels of protein—both conditions that cause disease.
Among the Amish, c. 1960s
Among the Amish, c. 1960s
Diana Kwon | Apr 30, 2018
Victor McKusick’s pioneering investigations provided insight into hereditary disorders.
Advisors to FDA Recommend Approval of Gene Therapy for Blindness
Advisors to FDA Recommend Approval of Gene Therapy for Blindness
Jef Akst | Oct 12, 2017
Spark Therapeutics’s Luxturna would be the first approved therapy in the U.S. that replaces or repairs a defective gene inherited from one’s parents.
Genotyping “Surrogates” Misdiagnoses Deadly Heart Condition
Genotyping “Surrogates” Misdiagnoses Deadly Heart Condition
Kerry Grens | Nov 2, 2016
Family members with a certain gene variant had unnecessary interventions, highlighting the potential hazards of precision medicine.