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Rare Disease-Focused-Ultragenyx Pharmaceutical Stock Falls On Serious Adverse Events In Eaarly Genetic Disorder Study
New data from Phase 1/2 study of GTX-102 for Angelman syndrome shows rapid and sustained improvement in patients, exceeding natural history data. read more.....»»
Vertex Pharmaceutical"s Move Beyond Its $10B Cystic Fibrosis Franchise, Pays Around $5B For Kidney Disease Player Alpine Immune
Vertex Pharma.....»»
Cartesian Therapeutics" Rare Disease Candidate Shows Long Lasting Benefit Over Argenx"s Vyvgart, Analyst Initiates With Buy
Cartesian Therapeutics' autoimmu.....»»
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.....»»
Vertex to study experimental gene-editing therapy as treatment for sickle cell, beta-thalassemia
Vertex Pharmaceuticals Inc. said Tuesday it plans to pay $1.1 billion to Crispr Therapeutics AG as part of a deal to test an experimental gene-editing therapy for sickle cell disease and transfusion-dependent beta-tha.....»»
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.....»»
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.....»»
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......»»
Translating passion into execution: Cure Rare Disease"s founder on the business of biotech
When a career path chooses you in the form of a pressing personal cause, passion and commitment must soon be followed by execution. And that means schooling up — quickly. I had heard the call of entrepreneurship early. I had a new Harvard B.....»»
SVB Leerink, J.P. Morgan downgrade Amicus stock after Phase 3 data for rare disease drug comes out
Shares of Amicus Therapeutics Inc. were down 23.1% in premarket trading on Friday, the day after the company said an experimental rare disease d.....»»
Chinook Therapeutics receives rare pediatric disease designation for CHK-336
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.....»»
Chinook Therapeutics Nabs Rare Pediatric Disease Tag For CHK-336 In Primary Hyperoxaluria
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Taysha gets both rare pediatric disease and orphan drug designations for TSHA-10
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.....»»
Seelos Therapeutics receives Rare Pediatric Disease Designation for SLS-005
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.....»»
The most common coronavirus symptoms in children — and how to know if they have a rare post-virus inflammatory syndrome
Getty Young people seem to be less vulnerable than older populations to the coronavirus, and tend to experience mild symptoms when they do get it. But reports of a poorly-understood and deadly inflammatory disease.....»»
Doctors in Italy have found a link between the coronavirus and the rare inflammatory disease seen in more than 100 children
Fiona Goodall / Stringer / Getty Images Doctors say there is evidence that a rare inflammatory syndrome being seen in children is connected to the novel coronavirus, according to a new study in .....»»