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6 days ago | 
rarediseaseadvisor.com | Ozge Ozkaya
Data for new and approved therapies for myelofibrosis (MF) were extracted, tabulated, and analyzed for clinical relevancy in a new article published in the scientific journal Hematological Oncology. “It remains to be seen if these newer agents can induce any remission in MF and enable patients to come off therapy, but the future is beginning to look much brighter,” wrote the researchers.
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6 days ago | 
rarediseaseadvisor.com | Ozge Ozkaya
Researchers from Turkey presented the case of a pediatric patient who had concomitant Hodgkin lymphoma and myelofibrosis (MF) who was successfully treated with a bone marrow transplantation. This is the first reported case of this treatment in such a context. “Pediatric myelofibrosis should be considered a differential diagnosis in children with [Hodgkin lymphoma],” the researchers wrote in a letter to the editor of the Turkish Journal of Haematology.
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6 days ago | 
rarediseaseadvisor.com | Ozge Ozkaya
Tadalafil treatment does not seem to have adverse effects on cardiovascular function in patients with Duchenne muscular dystrophy (DMD), according to a new study published in the journal BMC Cardiovascular Disorders. This finding is based on the results of echocardiograms, electrocardiograms, and measures of vital signs through 48 weeks. The study also showed that the treatment led to a small increase in the mean internal dimension and volume of the left ventricle.
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6 days ago | 
rarediseaseadvisor.com | Ozge Ozkaya
It is possible to reach minimal symptom expression in patients with chronic inflammatory demyelinating polyneuropathy (CIDP) following immunotherapy, according to a new study published in the journal Neurology. However, residual deficits are common.
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1 week ago | 
rarediseaseadvisor.com | Ozge Ozkaya
Researchers from Iran presented the case of a patient with chronic inflammatory demyelinating polyneuropathy (CIDP) who underwent emergency umbilical herniorrhaphy and whose anesthesia was successfully managed.
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1 week ago | 
rarediseaseadvisor.com | Ozge Ozkaya
A new study presented the case of 2 patients with hereditary neuropathy with liability to pressure palsies (HNPP) who were misdiagnosed. These cases show the importance of applying chronic inflammatory demyelinating polyneuropathy (CIDP) diagnostic criteria correctly, the authors said. Read more about the diagnosis of CIDPThe first patient was a 69-year-old male who presented with weakness and numbness in his legs that progressed slowly.
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1 week ago | 
rarediseaseadvisor.com | Ozge Ozkaya
Biomarkers in the blood can predict both current and future outcomes in patients with neuromyelitis optica spectrum disorder (NMOSD) and multiple sclerosis (MS), according to a new study published in the journal Scientific Reports. The study also found that these biomarkers have different patterns that represent the uniqueness of the pathogenesis of each disease.
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1 week ago | 
rarediseaseadvisor.com | Ozge Ozkaya
Patients with neuromyelitis optica spectrum disorder (NMOSD) who also have Sjogren’s disease have more severe disease at the time of attack and relapse earlier compared to those without Sjogren’s disease, according to a new study by researchers in China. The investigators also developed a nomogram that could predict the risk of NMOSD concomitant with Sjögren’s disease. Read more about NMOSD diagnosisThe study is published in the journal Arthritis Research and Therapy.
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1 week ago | 
rarediseaseadvisor.com | Ozge Ozkaya
Disease-modifying therapies are generally well-tolerated in patients with multiple sclerosis (MS) and related demyelinating diseases like neuromyelitis optica spectrum disorder (NMOSD), according to a real-world study published in the journal Therapeutic Advances in Neurological Disorders. The study also identified some atypical adverse events, enlarging the range of potential complications associated with disease-modifying therapies.
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1 week ago | 
rarediseaseadvisor.com | Ozge Ozkaya
An interventricular septum brightness index from the apical 4-chamber view of the heart may identify subtle calcifications in the septum and help differentiate between transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) and other types of cardiac amyloidosis, according to a new study published in the International Journal of Cardiology. A high brightness index may also have “prognostic value” in wild-type ATTR-CM, the authors said.
