Biotech companies working on gene therapies have made significant strides lately, but many of them remain relatively risky stocks. Still, gene therapies have the potential to help transform medicine by unlocking treatments or cures for illnesses that have so far eluded researchers.

This factor makes biotechs focused on gene therapies attractive targets for long-term investors, provided they pick the right stocks in this niche. With that said, let's look at one gene-therapy company that's been having a tough time of late, but is well worth investing in: Sarepta Therapeutics (NASDAQ: SRPT).

Sarepta focuses primarily (though not exclusively) on developing treatments for Duchenne muscular dystrophy (DMD), a genetic, progressive disease that weakens patients' muscles and leads to a shortened life expectancy. All of its four approved products target DMD. But the latest to earn approval from the U.S. Food and Drug Administration (FDA) is arguably the most important.

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Source Fool.com