Liver Complications Lead FDA to Stop Tests of Intellia Gene-Editing Therapy for a Rare Disease
The FDA has halted clinical testing of an Intellia Therapeutics gene-editing therapy following a report that a patient in a ...
Tag: Rare
Roche suffers a fresh blow as India’s Supreme Court allows generic sales of its pricey rare disease drug
In a case that was billed as profits versus patients, India’s Supreme Court dismissed a petition in which Roche sought ...
Sarepta to seek approval for gene therapy in rare muscular dystrophy
An experimental gene therapy from Sarepta Therapeutics increased levels of the gene missing in an ultra-rare form of muscular dystrophy, ...
Chiesi’s Rare Disease Strategy Expands to Genetic Meds via Alliance With CRISPR Startup Arbor
In the five years since Italy-based Chiesi Group established its rare disease division in Boston, the unit has landed regulatory ...
With FDA Nod in Rare Hormone Disease, Crinetics Can Challenge Blockbuster Meds From Novartis, Ipsen
Drugs for the rare hormone disorder acromegaly include older engineered peptides administered as frequent injections. A new FDA approval brings ...
CDC: Rare Salmonella strain from bearded dragons caused 2024 US outbreak, still circulates
Kent Kanouse / Flickr cc People who live with or handle pet bearded dragons are at continued risk for Salmonella ...
First Therapy for Rare Tumor Is a Blockbuster Prospect, But Does Precigen Have the Cash to Make It Happen?
Tumors caused by recurrent respiratory papillomatosis are benign, but that doesn’t mean they are not serious. In this rare disease, ...
In Crowded Market for a Rare Disease, Ionis Bets Patients Will Switch to Its New First-in-Class Drug
As with most rare diseases, the market for hereditary angioedema drugs is small. It’s also relatively crowded, served by several ...
FDA Turns Down Capricor Cell Therapy for Cardio Complications of Rare Muscle Disease
Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy has come up short at the FDA, with the regulator rejecting the ...
At ASCO, Merck KGaA Shows How Its Drug Measures Up as Treatment for Rare Type of Tumor
Patients with tenosynovial giant cell tumor can take some comfort in knowing that this rare type of tumor affecting joints ...
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