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New drugs are being developed to treat Duchenne Muscular Dystrophy — and many other rare diseases — but the treatments are stalled at the FDA. A new Kickstarter campaign is raising funds to complete a documentary about parents fighting to stop Duchenne Muscular Dystrophy and gain access to potentially life-saving drugs before the disease kills their children.
Fueled by new cancer therapeutics, last year the annual new molecular and biological entity approval count from the U.S. Food and Drug Administration (FDA) saw its highest year since 1997. One-third of the novel products approved by the FDA’s Center for Drug Evaluation and Research (CDER) are used to treat cancers of the blood, breast, colon, prostate, skin and thyroid.
