Rare Diseases

Orphan and rare diseases drug company Lobe Sciences Ltd. (OTCQB: LOBEF) is set to acquire Altemia & Company LLC. The deal includes all Altemnia assets, intellectual property, and commercial inventory to manage patients suffering from Sickle Cell Disease (SCD), a group of hereditary red blood...

The U.S. Food and Drug Administration (FDA) has granted PharmaTher Holdings Ltd. (OTCQB: PHRRF) an Orphan Drug designation for ketamine in the prevention of injury from organ transplantation. Orphan drug designation, which grants special status to a sponsor’s drug or biological product to...

The FDA has granted Rare Pediatric Disease Designation to Moleculin Biotech Inc's (NASDAQ: MBRX) WP1066 to treat ependymoma. Ependymoma is a rare type of tumor in the brain or spinal cord and most often occurs in young children. The drug candidate currently received...

Analysts at Ladenburg Thalmann turned incrementally bullish on Ignyta Inc (NASDAQ: RXDX) after the company's therapy for the treatment of rare lung cancer showed encouraging results in a preliminary clinical trial among patients with a defective cancer-causing gene that is found in around 2...

Shares of Amicus Therapeutics, Inc. (NASDAQ: FOLD) were trading higher by more than 12 percent Wednesday after the company released encouraging clinical trial data. The company's therapy showed a greater improvement in muscle and lung function for people suffering from Pompe, a rare disease...

Analysts at Morgan Stanley conducted a tracking survey to gain a better understanding of Sarepta Therapeutics Inc (NASDAQ: SRPT)'s outlook for its Exondys 51 therapy. After coming away with encouraging data, the firm's David Lebowitz upgraded Sarepta's stock rating from Equal Weight to Overweight...

Analysts at BTIG laid out the bullish case for owning shares of Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) — a "storied" company with a strong growth outlook. BTIG's Dane Leone initiates coverage of Vertex's stock with a Buy and $200 price target, on the thesis that...

Shares of Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) tumbled more than 12 percent Wednesday after the company offered a disappointing update concerning a muscle-disease drug study. The biopharmaceutical company, which focuses on the development of novel products for rare and ultra-rare diseases,...

Martin Shkreli, the so-called "most hated man on the Internet" is making news again. Retrophin Inc (NASDAQ: RTRX), a bio-pharmaceutical company that focuses on therapies for the treatment of rare diseases, saw its shares surge Wednesday. The company announced positive results from a...

Shire PLC (ADR) (NASDAQ: SHPG) on Tuesday proposed a combination with Baxalta Inc (NYSE: BXLT) in a $30 billion all-stock deal, implying a potential value of $45.23 per Baxalta share. Here is a roundup of what Wall Street's top analysts are saying. Berenberg: Deal EPS Accretive...