According to a recent article, investigators have proposed a checklist that will aid in determining the strength of potential gene-disease relationships for rare disease. The Need It has already…
April 15, 2022 marks the 9th annual International Pompe Day, designed to help raise awareness and education around Pompe disease. The slogan of International Pompe Day is "Together We…
According to a recent press release, the FDA has recently granted approval to Vijoice - also known under the generic name alpelisib - for the treatment of PIK3CA-related overgrowth spectrum…
A study recently published in the journal ESMO Open has documented new biomarkers which may be able to predict efficacy outcomes of antiangiogenic therapies (such as surufatinib) in patients diagnosed with neuroendocrine…
A recent study titled "Effect of FT218, a Once-Nightly Sodium Oxybate Formulation, on Disrupted Nighttime Sleep in Patients With Narcolepsy: Results From the Randomized Phase 3 REST-ON Trial," was published…
According to a recent article, last week was Porphyria Awareness Week, a week dedicated to recognizing and uniting those who are a part of the porphyria community. Porphyria Porphyria is…
If you have not heard of craniosynostosis, it is understandable, as the disorder affects only one infant out of approximately 2500. A recent article published in the Washington News…
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