Paroxysmal nocturnal hemoglobinuria (PNH) is a hematopoietic disorder with a nonspecific clinical presentation that often delays the diagnosis. We present the case of a 32-year-old female patient who ...

The paroxysmal nocturnal hemoglobinuria (PNH) market is projected to grow significantly by 2036, driven by emerging therapies and advancements in treatment options.

Hematuria is a hallmark clinical manifestation of IgA nephropathy (IgAN) and largely reflects underlying glomerular inflammation. Despite this, current guidelines prioritize proteinuria and estimated…

Hematuria is a hallmark clinical manifestation of IgA nephropathy (IgAN) and largely reflects underlying glomerular inflammation. Despite this, current guidelines prioritize proteinuria and estimated ...

The Paroxysmal Nocturnal Hemoglobinuria market is projected to grow from USD Billion 0.4377 in 2024 to USD Billion 0.8962 by 2035, at a CAGR of 6.73% during 2025-2035.

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Babesia aktasi is a recently discovered species that is highly prevalent in native goats in Türkiye’s Mediterranean region. Although it does not induce clinical disease in local breeds, it causes severe illness in Saanen goats, manifesting with high fever, anemia, hemoglobinuria, and jaundice. Imidocarb dipropionate (IMDP) has been reported to be therapeutically effective against Babesia species. This study aimed to evaluate the therapeutic efficacy of IMDP and its ability to eliminate the parasite in experimentally infected Saanen goats. Twelve goats were assigned to treatment and control groups (n = 5 per group) and infected using fresh blood from two splenectomized donors. All goats developed clinical babesiosis, with parasitemia ranging from 3.8% to 22. Semi-nested PCR specific to B. aktasi was performed for 30 days post-treatment. Following treatment with IMDP (1.2 mg/kg), clinical signs resolved by the third and fourth days, and parasitemia became microscopically undetectable. However, one goat in the treatment group and four goats in the control group died shortly after infection. Hematological parameters (HCT, RBC, HB) decreased during infection but normalized in surviving animals. Notably, B. aktasi DNA remained detectable by PCR up to 30 days after treatment. These findings indicated that IMDP resolved clinical signs and eliminated microscopically detectable parasitemia; however, it may not completely eliminate the parasite at the molecular level in B. aktasi infections. The results of this study provide valuable information for optimizing therapeutic approaches and improving our understanding of treatment responses in new species B. aktasi.

Babesia aktasi is a recently discovered species that is highly prevalent in native goats in Türkiye’s Mediterranean region. Although it does not induce clinical disease in local breeds, it causes severe illness in Saanen goats, manifesting with high fever, anemia, hemoglobinuria, and jaundice. Imidocarb dipropionate (IMDP) has been reported to be therapeutically effective against Babesia species. This study aimed to evaluate the therapeutic efficacy of IMDP and its ability to eliminate the parasite in experimentally infected Saanen goats. Twelve goats were assigned to treatment and control groups (n = 5 per group) and infected using fresh blood from two splenectomized donors. All goats developed clinical babesiosis, with parasitemia ranging from 3.8% to 22. Semi-nested PCR specific to B. aktasi was performed for 30 days post-treatment. Following treatment with IMDP (1.2 mg/kg), clinical signs resolved by the third and fourth days, and parasitemia became microscopically undetectable. However, one goat in the treatment group and four goats in the control group died shortly after infection. Hematological parameters (HCT, RBC, HB) decreased during infection but normalized in surviving animals. Notably, B. aktasi DNA remained detectable by PCR up to 30 days after treatment. These findings indicated that IMDP resolved clinical signs and eliminated microscopically detectable parasitemia; however, it may not completely eliminate the parasite at the molecular level in B. aktasi infections. The results of this study provide valuable information for optimizing therapeutic approaches and improving our understanding of treatment responses in new species B. aktasi.

Nuvectis Pharma (NASDAQ: NVCT)/b) reported that ciprocopan (NXP100), a once-daily oral Complement Factor B inhibitor, received marketing approval from China’s NMPA for treating Paroxysmal Nocturnal Hemoglobinuria (PNH) patients previously untreated with complement inhibitors.This is the first global approval of a once-daily oral Factor B inhibitor and is based on a head-to-head Phase 3 trial versus Soliris in treatment‑naive PNH patients, where ciprocopan met all primary and secondary endpoints, showed superior hemoglobin responses and transfusion avoidance, and had no discontinuations due to adverse events. Haisco developed and controls ciprocopan in China, while Nuvectis holds exclusive rights outside Greater China, India and parts of Southeast Asia.