14 Table 1 Data on Subjects with Homozygous Deficiency of Alpha1-Antitrypsin ... Showing that the Correlation between the Quantity of Alpha 1-Antitrypsin (Abscissa) and the Total Antitryptic ...

Shares in Wave Life Sciences rose by 74% after it reported clinical data with a pioneering new RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD). Wave claims this is the first clinical ...

Krystal Biotech KRYS reported fourth-quarter earnings per share of $1.52, which beat the Zacks Consensus Estimate of $1.29 as ...

Baron Funds, an investment management company, released its “Baron Health Care Fund” fourth quarter 2024 investor letter. The ...

Q4 2024 Management View CEO Krish Krishnan highlighted 2024 as a strong year, driven by the continued success of the VYJUVEK launch in the U.S., which is tracking as a top-tier rare disease launch. He ...

which is in Phase 1 clinical trials for treating Alpha-1 antitrypsin deficiency; and KB301 that is in Phase 2 clinical trials for treating aesthetic skin conditions, as well as in open label study ...

NEW YORK, NY / ACCESS Newswire / February 20, 2025 / If you suffered a loss on your Intellia Therapeutics, Inc. (NASDAQ:NTLA) investment and want ...

NEW YORK, NY / ACCESS Newswire / February 18, 2025 / If you suffered a loss on your Intellia Therapeutics, Inc. (NASDAQ:NTLA) investment and want ...

BioMarin Pharmaceutical Inc. ( NASDAQ: BMRN) Q4 2024 Earnings Conference Call February 19, 2025 4:30 PM ET Traci McCarty - Head of IR Alexander Hardy - President and CEO Brian Mueller - EVP and CFO ...

Rosen Law Firm, a global investor rights law firm, announces that a shareholder filed a class action on behalf of purchasers of securities of Intellia Therapeutics, Inc. between July 30, 2024 and ...