rare diseases
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News
APLASTIC ANEMIA | Incidence and Clinical Management in Spain Consistent with Other Studies
The study was carried out through a multicenter, ambispective, observational approach encompassing 7 Spanish hospitals, catering to roughly 3.91 million individuals, over the span from 2010 to 2019. The research…
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NEW TREATMENTS | Gene Editing, mRNA Hold Promise as Fanconi Anemia Therapies
That is the major finding of a newly published review that examined how a better understanding in the scientific and molecular underpinnings of the disease has translated into potential new…
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NEW APPROVAL | Health Canada Grants Approval To Sobi’s PNH Therapy
The treatment is indicated for such patients with insufficient response to, or are not tolerant to, a C5 inhibitor. The latest development is based on the findings from the head-to-head…
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RARE ANAEMIAS | Shining a Spotlight on Rare Anaemias’ Education and Advocacy with RAIN
Recognising the absence of a global umbrella organisation for rare anaemias to advocate and safeguard the rights of patients in a consistent way, and the commonalities between rare anaemias in…
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SCIENCE NEWS | Research On Rare Genetic Disease Sheds Light On A Common Head And Neck Cancer
Patients with the rare genetic disorder Fanconi anemia lack certain elements of this repair system, making their cells incapable of removing lesions created by DNA-damaging chemicals, called aldehydes. And…
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PROFILE | After Major Advances In Hematology, Dr David Bodine Retires
When David M. Bodine, Ph.D., arrived at the US National Institutes of Health (NIH) for a postdoctoral fellowship in Arthur Nienhuis’s laboratory in the National Heart, Lung and Blood Institute (NHLBI),…
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DIAGNOSTICS | FDA Clears Baebies Disposable Enzyme Deficiency G6PD Test
Using a 50 µL blood sample (one drop of blood), FINDER delivers results approximately 15 minutes after sample introduction. Test results display G6PD enzyme activity in units per gram…
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TIF At The NORBS Online Regional Conference For Rare Diseases – 23 0ctober 2020
Supporting Serbia and other Eastern European countries in their efforts to upgrade holistic care services for patients with rare diseases, including thalassemia, TIF’s Executive Director, Dr Androulla Eleftheriou, participated on…
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