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Tackling deadly newborn disease, BridgeBio affiliate wins FDA drug approval
The BridgeBio model is designed to move drugs tackling rare diseases with a well-defined genetic basis quickly and efficiently to approval......»»
Quench Bio launches with $50M to treat "missing link" to inflammatory disease
What do fatty liver disease, asthma and a rare genetic condition called Familial Mediterranean Fever have in common? A newly launched biotech company believes they can all be treated with one approach......»»
The Daily Biotech Pulse: Osmotica Soars On Insider Buys, Ultragenyx"s Partnered Rare Disease Drug Moves Into Clinics
Here's a roundup of top developments in the biotech space over the last 24 hours. Scaling The Peaks (Biotech Stocks Hitting 52-week highs on Jan. 15) 0 read more.....»»
Ultragenyx"s (RARE) UX007 Gets Fast Track Status by FDA
Ultragenyx???s (RARE) UX007 receives Fast Track designation and Rare Pediatric Disease designation by the FDA for the treatment of long-chain fatty acid oxidation disorders.....»»
Novartis buying AveXis for $8.7B in big gene therapy bet
AveXis has been working on a treatment for babies with a rare genetic disease AveXis has been working on a treatment for babies with a rare genetic disease .....»»
Novartis buying gene therapy company AveXis for $8.7 billion
AveXis has been working on a treatment for babies with a rare genetic disease AveXis has been working on a treatment for babies with a rare genetic disease .....»»
Rare disease drug startup that raised $64 million a few weeks ago now seeks $115 million IPO
Eidos Therapeutics has just one experimental drug in a rare, fatal genetic disease, but it has moved the drug into the clinic quickly......»»
Cambridge"s Akcea rebounds with FDA approval of first drug
After having one of its drugs rejected by the FDA in August, Cambridge biotech Akcea Therapeutics Inc. turned the tide on Friday, receiving approval for another treatment for a rare genetic disease. The FDA approved Tegsedi, known generically as ino.....»»
Cartesian Therapeutics" Rare Disease Candidate Shows Long Lasting Benefit Over Argenx"s Vyvgart, Analyst Initiates With Buy
Cartesian Therapeutics' autoimmu.....»»
Ultragenyx announces longer-term data from Glycogen Storage Disease study
See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»
ChemoCentryx"s stock dives after critique from the FDA about experimental treatment for rare autoimmune disease
Shares of ChemoCentryx Inc. plunged 44.8% in trading on Tuesday after the Food and Drug Administration questioned the rat.....»»
Bluebird Bio provides update on severe genetic disease programs and business ops
See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»
Ultragenyx Pharmaceutical Inc (RARE) EVP and Chief Medical Officer Camille L Bedrosian Sold ...
Related Stocks: RARE,.....»»
FibroGen receives rare pediatric disease designation from FDA for pamrevlumab
See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»
Moleculin Biotech"s WP1066 awarded Rare Pediatric Disease Designation by FDA
See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»
Moleculin Biotech Stock Is Trading Higher After WP1066 Scores Rare Pediatric Disease Tag For Type Of Brain Tumor
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Akouos receives orphan drug and rare pediatric disease designations for AK-OTOF
See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»
Ultragenyx, n-Lorem Foundation team on ultra-rare diseases treatment development
See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»
Ionis shares tumble 19% after Roche halts Phase 3 study for rare-disease drug
Shares of Ionis Pharmaceuticals Inc. were down 19.6% in premarket trading on Tuesday, the day after the company said Roche Holding AG decided to discontinue.....»»
Ionis shares tumble19% after Roche halts Phase 3 study for rare-disease drug
Shares of Ionis Pharmaceuticals Inc. were down 19.6% in premarket trading on Tuesday, the day after the company said Roche Holding AG decided to discontinue.....»»