Duchenne Muscular Dystrophy

Shares of Sarepta Therapeutics Inc (NASDAQ: SRPT) are tanking Tuesday morning as a binary event turned up a negative outcome for the company. What Happened Sarepta announced Monday after the market close the FDA issued a complete response letter for its NDA seeking accelerated approval for...

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced Friday that the Food and Drug Administration approved a label expansion for the drug Emflaza to include younger patients with Duchenne muscular dystrophy who are between 2 and 5 years old.  PTC Therapeutics shares were trading...

The microcap biotech Solid Biosciences Inc (NASDAQ: SLDB)'s shares were losing more than one-quarter of their value Tuesday after the company issued a clinical trial update on its Duchenne muscular dystrophy gene therapy candidate with its first-quarter earnings release. What Happened...

Small-cap biotech Wave Life Sciences Ltd (NASDAQ: WVE) — which fell last week following a delay in the release of top-line data from its Huntington disease candidates — is once again under pressure Tuesday. What Happened Wave Life Sciences released final results Tuesday...

The Street grew more bullish on Solid Biosciences Inc (NASDAQ: SLDB) last week following progress announcements on Solid and peer candidates. At least three analysts raised their valuation targets: The Rating Chardan analyst Gbola Amusa maintained a Buy rating on the stock, but increased his price...

Sarepta Therapeutics Inc. (NASDAQ: SRPT) reported some encouraging results from the first three patients treated with the company’s microdystrophin gene therapy program for Duchenne Muscular Dystrophy (DMD), sending the stock soaring higher by 40 percent Tuesday and prompting positive...

Sarepta Therapeutics Inc (NASDAQ: SRPT) shares, halted shortly before Tuesday's open, opened higher by more than 50 percent. What Happened Sarepta released Phase 1/2 data on its microdystrophin gene therapy for Duchenne muscular dystrophy, or DMD, which showed robust levels of microdystrophin...

Multiple small- and mid-cap biotech stocks have benefited from a strong run, prompting Morgan Stanley to downgrade three companies in the group. The Analyst Morgan Stanley's Matthew Harrison downgraded the following: Sarepta Therapeutics Inc (NASDAQ: SRPT) from Overweight to Sector Weight...

The young biotech firm Solid Biosciences Inc (NASDAQ: SLDB) secured two Buy ratings Tuesday and another Wednesday. The Rating Chardan Capital analyst Gbola Amusa initiated Solid Biosciences with a Buy rating and $40 price target. The Thesis Amusa expects gene therapies to penetrate 26.8...

Just weeks into its life as a publicly traded company, Solid Biosciences Inc (NASDAQ: SLDB) caught the eyes of four analysts who assumed coverage Tuesday with a mix of Holds and Buys. Here is what one of the more bearish critics had to say. The Rating Goldman Sachs analyst Salveen Richter...

Shares of Sarepta Therapeutics Inc (NASDAQ: SRPT) gained more than 1 percent on Thursday after the company reported its third consecutive beat and raise earnings report, analysts at Cowen said in a research report. The firm's Ritu Baral,/a> maintains an Outperform rating on Sarepta's stock with an...

Shares of PTC Therapeutics, Inc. (NASDAQ: PTCT) gained more than 13 percent Friday after the FDA found that the company's experimental drug to treat Duchenne muscular dystrophy may work. The FDA's Advisory Committee meeting ruled that the body of evidence for the company's ataluren...

Shares of Sarepta Therapeutics Inc (NASDAQ: SRPT) soared by more than 10 percent Wednesday after the company reported encouraging results from a clinical trial of a therapy intended to treat Duchenne muscular dystrophy (DMD). Sarepta's drug called golodirsen showed that patients who suffer from...

Sarepta Therapeutics Inc (NASDAQ: SRPT), a smid-cap biopharmaceutical company that focuses on the treatment of rare neuromuscular diseases including Duchenne muscular dystrophy, saw its stock peak north of $60 per share in late 2016 after the U.S. Food and Drug Administration green-lighted its DMD...

Even as it recovered from poor fourth-quarter results, Capricor Therapeutics Inc (NASDAQ: CAPR) pumped a little hope into the world of Duchenne muscular dystrophy last week with positive clinical results from its CAP-1002 study. And the breadth of impact could ultimately be more expansive. “...