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Study shows how gene therapy may help protect against vision loss in multiple sclerosis

Study shows how gene therapy may help protect against vision loss in multiple sclerosis

Research findings may one day help keep ALS at bay

Research findings may one day help keep ALS at bay

AAD honors board-certified dermatologists for treatment of a patient with aquagenic keratoderma

AAD honors board-certified dermatologists for treatment of a patient with aquagenic keratoderma

Better definition could lead to better diagnosis, treatment for rare diseases

Better definition could lead to better diagnosis, treatment for rare diseases

Research team publishes findings from study of TAF1 syndrome

Research team publishes findings from study of TAF1 syndrome

PCORI Board approves $70 million to support projects designed to improve care for health conditions

PCORI Board approves $70 million to support projects designed to improve care for health conditions

Tailored T-cells safe, may be effective in preventing and treating multiple viral infections

Tailored T-cells safe, may be effective in preventing and treating multiple viral infections

Newly identified antibody has potential to stop, reverse pulmonary arterial hypertension

Newly identified antibody has potential to stop, reverse pulmonary arterial hypertension

Study sheds light on how Zika virus causes microcephaly in newborns

Study sheds light on how Zika virus causes microcephaly in newborns

UM launches CAR-T clinical trial for patients with myasthenia gravis

UM launches CAR-T clinical trial for patients with myasthenia gravis

FDA keeps brand-name drugs on a fast path to market ― despite manufacturing concerns

FDA keeps brand-name drugs on a fast path to market ― despite manufacturing concerns

Molecular gatekeepers that regulate calcium ions vital for muscle health and repair

Molecular gatekeepers that regulate calcium ions vital for muscle health and repair

Discovery in monkeys could pave way for treatment of Bardet-Biedl Syndrome

Discovery in monkeys could pave way for treatment of Bardet-Biedl Syndrome

Breakthrough study suggests ways to develop treatments for rare genetic skin disease

Breakthrough study suggests ways to develop treatments for rare genetic skin disease

$5 million in federal funding to accelerate potential treatments for urea cycle disorders

$5 million in federal funding to accelerate potential treatments for urea cycle disorders

Study: More than 300 million people affected worldwide by rare diseases

Study: More than 300 million people affected worldwide by rare diseases

Hong Kong Baptist University receives FDA orphan drug designation for novel aptamer

Hong Kong Baptist University receives FDA orphan drug designation for novel aptamer

LSU Health New Orleans awarded $1.74 million to advance research on Usher syndrome

LSU Health New Orleans awarded $1.74 million to advance research on Usher syndrome

Cytel hosts Complex Innovative Trial Design Symposium and East Training in Boston

Cytel hosts Complex Innovative Trial Design Symposium and East Training in Boston

Study suggests new model of treatment for heart attack or stroke victims

Study suggests new model of treatment for heart attack or stroke victims