The FDA evaluates not only a single measurement, but the entire data package from the ST-920 study conducted by $SGMO. Although the eGFR (estimated glomerular filtration rate = measure of kidney function) is a particularly important individual value, it is not the only criterion.

I keep asking myself, is $SGMO's GirFit really worthless? A patient with severe hemophilia A and active inhibitors who had been receiving #PFE marstacimab for prevention for approx. three years died on December 14, 2025 following a thrombotic stroke after minor surgery. This

$PFE reports #DEATH in #HYMPAVZI #THROMBOTIC #STROKE

$PFE reported #DEATH in hympavzi Hemophilia A

As the year comes to an end Im still waiting for a Fabry deal for $SGMO .46 which will give them plenty of cash & upside - FDA approval should happen with its superior data-with last weeks $4.8 billion acquisition of $FOLD we now have a bench mark of what Fabry disease is worth

BREAKING $PFE PFIZER REPORTS #DEATH IN #HYMPAVZI (marstacimab) of a HEMOPHILIA A patient PATIENT DIED OF #THROMBOTIC #STROKE ! HYMPAVZI ist the new drug from pfizer they want to sell (once weekly) lifelong to patients... for that they stopped ONCE ONLY $SGMO Hemo A GT + B GT

Towards a better definition of left ventricular hypertrophy in Fabry disease: sex-specific thresholds to personalize prognostication https://t.co/h19NWVPyJa Stable cardiac function was observed with $SGMO s ST-920 over one and two years!!

$EDIT $CRSP $NTLA $BEAM $SGMO $LLY Gene editing is considered a revolutionary approach for genetic heart diseases and could, in the long term, enable curative therapies rather than merely symptomatic treatments. However, further research, technological improvements, and safety

$SGMO case study from Sangamo to be presented in April At #ASIA conference you can see every other presenation has a #NAMED presenter, but this one ... who will present this in ASIA ? #Astellas ?

The Commissioner’s National Priority Voucher (CNPV) Pilot Program is a new pilot program launched by the FDA in which selected drug developments can receive a voucher that enables a greatly accelerated review process (1–2 months instead of 10–12 months) and closer communication

A specific list price for Elfabrio in Canada has not yet been published in any accessible regulatory or media reports. For enzyme-specific treatments such as Fabrazyme or Replagal, the annual cost in Canada was approximately $300,000 per patient, which could be a realistic

For similar enzyme replacement therapies for Fabry disease (e.g. agalsidase alfa or beta), the annual treatment costs in Canada or under the PharmaCare system are approximately $299,000–$311,000 per year (based on the Canadian PharmCare overview; figures are in US dollars and

Health Canada approved the enzyme replacement therapy Elfabrio (pegunigalsidase alfa) to treat adults with Fabry disease. https://t.co/QdItqfUGLF After 12 months of treatment with pegunigalsidase alfa, the average eGFR value is negative, namely: -1.19 mL/min/1.73 m²/year. $SGMO

The Phase 1/2 study showed a positive mean annual eGFR slope (a measure of kidney function) of approximately 1.97 to 3.06 mL/min/1.73 m² per year, indicating stabilization or improvement in kidney function. These data are particularly important because eGFR slope is a recognized

The consequences of Fabry disease can have a lasting impact on life: kidney and heart failure, leading to dialysis or kidney and/or heart transplantation. Regardless of whether $SGMO achieves economic success as a result, I consider it commendable to contribute to the financing

$SGMO Fabry disease is a rare, hereditary metabolic disorder (lysosomal storage disease). Due to a deficiency or absence of the enzyme α-galactosidase A, Gb3 (globotriaosylceramide) accumulates in many organs—the heart and kidneys are particularly affected. Cardiac involvement is

Although Fabry disease is progressive, columnist Jerry Walter does what he can to maintain optimal health and improve his outcome. However, the list of limitations is extensive—only gene therapy with $SGMO ´s ST-920 appears to offer a genuine remedy. https://t.co/VEtEBkataG

$CGEN secured a $65M upfront payment from $AZN, with up to $25M more tied to BLA milestones, by monetizing part of its royalty interest in rilvegostomig while retaining the majority of future royalties and upside

$VERU received FDA clarity supporting enobosarm plus GLP-1 RA as a combination obesity therapy, with incremental fat loss accepted as a primary endpoint; following positive ph2b QUALITY results showing preserved lean mass and physical function, Veru will initiate the ph2 PLATEAU

$QURE disclosed that the FDA does not view current phI/II data for AMT-130 in Huntington’s disease as sufficient to support a BLA, clouding the program’s regulatory path.