Non-transfusion-dependent thalassemia encompasses three clinically distinct forms -- beta-thalassaemia intermedia, hemoglobin E/beta-thalassaemia (mild and moderate forms), and al ...

Q4 2024 Earnings Call Transcript February 10, 2025 Vertex Pharmaceuticals Incorporated misses on earnings expectations. Reported EPS is $3.98 EPS, expectations were $4.02. Operator: Good day, and ...

Their flagship product, Gazelle, is an affordable point-of-care diagnostic tool that uses electrophoresis to test for a variety of diseases including sickle cell and beta thalassemia ... For ...

In particular, patients suffering from severe disease (beta thalassemia major ... compared to 1.6% in the placebo group (p<0.0001; See pg 22/23 from Agios 2024 Q3 10-Q). Furthermore, patients ...

CRISPR booked revenue of just under $36 million. On the bottom line, CRISPR flipped to a generally accepted accounting ...

The therapy involves taking stem cells out of a patient's bone marrow and editing a gene in the cells in a laboratory and ...

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the ...

Reshma Kewalramani; President, Chief Executive Officer, Director; Vertex Pharmaceuticals Inc Stuart Arbuckle; Chief Operating Officer, Executive Vice President; Vertex Pharmaceuticals Inc Charles ...

The gene therapy Casgevy, which will now be offered to some patients in England, could help people live disease-free, experts ...

Some you might hold only temporarily, while others you own for decades. David Jagielski (Eli Lilly): A forever stock you can ...

Ongoing launch of CASGEVY ® continues to gain momentum; new cell patient collection initiations expected to significantly grow in 2025- -More than 50 authorized treatment centers (ATCs) activated ...