BioSpace1d
Cystic Fibrosis Biotech Sionna Announces $150M IPO
The Massachusetts-based biotech plans to use the funds to push its candidates into mid-stage clinical trials in a space ...
Armed robbery in Revesby2d
Enid's Cystic Fibrosis Clinical Trial Offers Hope
By the time Enid Katz was two years old, she had already suffered through several bouts of bronchitis and was always hungry, ...
pharmaphorum6d
Vertex gets EU okay to treat younger cystic fibrosis patients
A new EMA approval has expanded the use of Vertex Pharma’s cystic fibrosis therapy Symkevi ... other so-called “minimal function” mutations in CFTR. The new EU approval comes after the ...
Is Vertex Pharmaceuticals a Buy Now That the FDA Approved Its New Pain Drug?
Lately, one of America's leading pharmaceutical companies can't catch a break from Wall Street. Shares of Vertex ...
C&EN6d
Discovery and Development of CFTR Modulators for the Treatment of Cystic Fibrosis
Article Views are the COUNTER-compliant sum of full text article downloads since November 2008 (both PDF and HTML) across all institutions and individuals. These metrics are regularly updated to ...
AOL3d
Is Vertex Pharmaceuticals a Buy Now That the FDA Approved Its New Pain Drug?
In December, the FDA approved Alyftrek, a new once-daily pill for patients with cystic fibrosis caused by at least one of 303 possible mutations in their CFTR genes. Alyftrek's precursor ...
Cystic Fibrosis Foundation4d
About Cystic Fibrosis
In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional. When the protein is not working correctly, it’s ...
SciELO2d
A new era of cystic fibrosis therapy with CFTR modulators
Cystic Fibrosis ... 1 Mechanism of action and CFTR variants approved by the US Food and Drug Administrations and the Brazilian National Health Surveillance Agency. Modulators Action Mechanism FDA ...
FierceBiotech2d
Sionna eyes up $156M IPO to fund cystic fibrosis trials
Sionna Therapeutics is eyeing a $156 million IPO as the cystic-fibrosis-focused biotech looks to take its lead candidate into ...
Seeking Alpha4d
VRTX Vertex Pharmaceuticals Incorporated
Vertex Pharmaceuticals Incorporated, a biotechnology company, engages in developing and commercializing therapies for treating cystic fibrosis (CF ... includes VX-522, a CFTR mRNA therapeutic ...
Nature1d
Diagnosis and management of familial Mediterranean fever: Integrating medical genetics in a dedicated interdisciplinary clinic
In the event that two mutations are not detected by either targeted mutation analysis or full gene sequencing in an individual with high clinical suspicion for FMF, another helpful diagnostic ...
Nature2d
Mechanisms of change in gene copy number
The locus-specific mutation frequencies for copy number variation and other structural changes are two to four orders of magnitude greater than for point mutations. HR mechanisms generally achieve ...