A recent study documenting cases of pregnancy in individuals living with paroxysmal nocturnal hemoglobinuria (PNH) was published in the Saudi Journal of Medicine & Medical Sciences. Previously, research on pregnancy with…
In a recent news release, rare disease-focused biopharmaceutical company CANbridge Pharmaceuticals Inc. ("CANbridge") shared that the National Medical Products Administration (NMPA) of China accepted an Investigational New Drug (IND) application…
As reported recently in Medscape, forty-eight patients with myotonic dystrophy type 1 (DM1), were identified by way of the DM-Scope registry, which is the largest collection of data for the DM population. The…
Renal cell carcinoma (RCC) is typically treated with single-agent tyrosine kinase inhibitors (TKIs). Now, this may be changing as research demonstrates the potential of the combination of TKIs and immunotherapy.…
“I’ll be honest with you, Lindsey. I had to look up your syndrome before this appointment,” the doctor bashfully admitted. “I have never seen or heard of CLOVES syndrome.” Though…
On August 2, 2021, biotechnology company Amicus Therapeutics ("Amicus") shared that its oral treatment Galafold (migalastat) was approved by the European Commission (EC) for the treatment of adolescent patients (ages…
A new study published in the journal Scientific Reports has helped explain the differences between neuromyelitis optics spectrum disorder (NMOSD) after optic neuritis (ON) and multiple sclerosis (MS). Although previous studies have…
According to a press release from late July 2021, LX1004, a treatment for patients with CLN2 Batten disease, received both Orphan Drug and Rare Pediatric Disease designations from the…
Clinical guidelines are often used to improve patient treatment. These guidelines use evidence reviews, as well as an examination of other standards-of-care, to optimize patient care and outcomes. According…
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