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@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
US POMPE DISEASE DISEASE LANDSCAPE

Roughly 70-80% of the 5,000-10,000 US Pompe patients have late-onset disease, yet years often pass before a blood-spot test confirms it. Pompe disease is an...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
UK POMPE DISEASE DISEASE LANDSCAPE

Roughly a quarter of UK Pompe patients on long-term ERT are inadequate responders, the target population for avalglucosidase alfa. Pompe disease is caused by acid...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
GCC POMPE DISEASE DISEASE LANDSCAPE

Saudi newborn screening will detect 25-35 infantile Pompe cases yearly, while late-onset patients are still found at 55-65% FVC. Pompe disease incidence in the GCC...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
US PNH DISEASE LANDSCAPE

PNH's 800-1,200 patient EVH subpopulation, not the full 15,000-20,000 prevalence estimate, defines the complement-inhibitor era opportunity. PNH is a clonal...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
UK PNH DISEASE LANDSCAPE

UK PNH diagnosis takes just 6-12 months through Leeds, yet 30% of the ~600 tracked patients also have aplastic anaemia. PNH is a clonal haematopoietic stem cell...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
GERMANY PNH DISEASE LANDSCAPE

Germany's own DGHO guideline admits it has no national PNH data, borrowing 16 cases per million from UK and France. PNH is a clonal haematopoietic stem cell...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
GCC PNH DISEASE LANDSCAPE

Fewer than 15 GCC labs run FLAER flow cytometry, holding confirmed PNH cases below 400 against 2,000-3,000 estimated patients. Paroxysmal nocturnal hemoglobinuria...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
FRANCE PNH DISEASE LANDSCAPE

France's own hospitalization database puts 2022 PNH prevalence near 1 in 94,000, below Germany's borrowed 16-per-million figure. A 2025 real-world study drawn from...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
US OBESITY DISEASE LANDSCAPE

41.9% of US adults meet the obesity threshold, but comorbidity segments, not the headline count, decide who gets covered. Obesity is a chronic, relapsing metabolic...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
US NON-SMALL CELL LUNG CANCER DISEASE LANDSCAPE

NSCLC splits into five biomarker-defined segments, and 51% of US patients are diagnosed only after the disease reaches distant stage. Non-small cell lung cancer is...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
US MYASTHENIA GRAVIS DISEASE LANDSCAPE

Myasthenia gravis is stratified by antibody subtype, not severity; only the roughly 85% of US patients who are AChR-positive can access C5 inhibitors. Myasthenia...

@moatrx • 2 weeks ago • comments: 0

Posted a new blog:
UK MYASTHENIA GRAVIS DISEASE LANDSCAPE

Efgartigimod's June 2025 NICE rejection (TA1069) leaves roughly 4,000 UK generalised MG patients without a recommended novel agent. Myasthenia gravis is an...

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