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Bluebird Bio Gene Therapy Shows Early Promise in Fatal Childhood Brain Disease

Bluebird is trying to use a gene therapy to correct the rare and fatal genetic defect found in boys with CALD.

Chiasma Craters on FDA Drug Rejection
Chiasma Craters on FDA Drug Rejection

04/18/16 - 08:46 AM EDT

Chiasma shares are plunging Monday following the U.S. Food and Drug Administration rejection of the company's drug to treat acromegaly, a rare disease of the pituitary gland.

Loxo Cancer Drug Targets Gene Mutation With Durable Responses in Early Study

Six patients, all with TRK fusion cancers unresponsive to current therapies, reported tumor shrinkage following treatment with Loxo's drug LOXO-101.

Biotech Stock Mailbag: Sarepta Therapeutics' Big Event Is Coming

TheStreet's Adam Feuerstein answers reader questions about biotech investing. There were a high number of emails on the upcoming Sarepta FDA advisory panel.

Chiasma's Acromegaly Drug Headed for FDA Rejection, Investor Says

Chiasma is expecting the U.S. Food and Drug Administration to make an approval decision Friday on a new oral treatment for acromegaly, a rare disease of the pituitary gland.

Clovis Lung Cancer Drug Trashed at FDA Panel, CEO Exit Next?

An FDA advisory committee delivers a brutal beatdown of Clovis' lung cancer drug rociletinib. It could cost Clovis' CEO his job.

Amicus Shares Barely Budge on Prediction of $3 Billion Buyout

Investors give the side eye to a prediction from Chardan analyst Gbola Amusa that Amicus Therapeutics will be acquired for three times its current market value

Anavex Plays the Orphan Drug Stock Promotion Game

Too many investors have a fundamental misunderstanding about the significance of orphan drug designation. I should say, insignificance, because the FDA delineation, on its own, means very little.

Endo International Tumbles as Insys Expects Weak Painkiller Sales

Shares of Endo fall as much as 9% Monday in sympathy with Insys Therapeutics, which pre-announced sharply weaker first-quarter sales of its fentanyl painkiller Subsys.

Allergan Could Buy These Drug Companies Now That the Pfizer Merger Is Scuttled

Now that the Allergan-Pfizer merger is off, Allergan CEO Saunders could start shopping again for biotech and smaller pharma companies to fuel his company's growth.

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