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@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
UK FABRY DISEASE LAUNCH READINESS

The £144M NHS Fabry market already has two established agents, so only an ADA-positive or female-heterozygote niche clears NICE. Agalsidase beta (Fabrazyme,...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
GCC FABRY DISEASE LAUNCH READINESS

Both approved Fabry ERTs are already NPHC-covered in the GCC, so the opening is the 30 to 50 patient ADA-positive cohort no one serves. Unusually for GCC rare...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
US DRAVET SYNDROME LAUNCH READINESS

Refractory Dravet is a 1,400 to 2,000-patient US market, and the clinical bar is over 40% additional seizure reduction beyond CBD plus fenfluramine. Cannabidiol...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
UK DRAVET SYNDROME LAUNCH READINESS

600 to 900 UK Dravet patients remain inadequately controlled on CBD plus fenfluramine combined, and a cardiac-monitoring-free profile is worth real NHS money....

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
GCC DRAVET SYNDROME LAUNCH READINESS

Cannabidiol is a Schedule 1 equivalent narcotic across the GCC, so clearing the SFDA Controlled Drug Board matters more than efficacy. Cannabidiol (Epidiolex) is...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
US COLD AGGLUTININ DISEASE LAUNCH READINESS

Sutimlimab left 46 percent of trial patients without a hemoglobin response, yet two rival complement programs abandoned the disease after its launch. Sutimlimab...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
US HR-POSITIVE HER2-NEGATIVE BREAST CANCER LAUNCH READINESS

A 2025 study of 9,146 patients found no survival difference across three first-line CDK4/6 inhibitors, closing that line to new entrants. A 2025 Flatiron Health...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
US ATTR AMYLOIDOSIS LAUNCH READINESS

Capturing newly diagnosed ATTR-CM volume, not switching stable tafamidis patients, is the path past ICER's steepest value gap in rare disease. Tafamidis (Vyndaqel)...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
UK ATTR AMYLOIDOSIS LAUNCH READINESS

Any new ATTR-CM stabiliser is judged against tafamidis's NICE TA984 QALY model, while 15,000 to 36,000 UK patients remain undiagnosed. Tafamidis (Vyndaqel 61mg,...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
GCC ATTR AMYLOIDOSIS LAUNCH READINESS

No ATTR-CM treatment is SFDA-registered in the GCC, so a first-mover drug still has almost no diagnosed patients without Tc-PYP expansion. Tafamidis (Vyndaqel) is...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
GCC DRAVET SYNDROME KOL MAPPING

A five-institution Saudi consortium spanning three cities tracked 44 Dravet syndrome patients on stiripentol combination therapy. GCC Dravet syndrome expertise...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
US HR+/HER2- METASTATIC BREAST CANCER KOL MAPPING

A 9,146-patient Flatiron Health study found no survival difference among palbociclib, ribociclib, and abemaciclib, so KOL influence decides prescribing share. The...

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