Mepsevii (vestronidase alfa) is a treatment for Mucopolysaccharidosis VII (MPS VII). The Committee for Medicinal Products for Human Use (CHMP) has given a positive recommendation for the marketing authorisation of…
When others learn that you are the parent of a child with a rare disease, they most likely correctly assume that doctor appointments and hospitals might be involved. Yet few…
Collaboration between the FDNA, the Cure Sanfilippo Foundation, and the Jonah’s Just Begun Foundation has led to technology that successfully recognises the facial phenotype (observable characteristics) of patients with mucopolysaccharidosis…
A gene therapy being developed as a treatment for Sanfilippo syndrome has just been awarded Regenerative Medicine Advanced Therapy (RMAT) status in the US, reports GlobalGenes. Sanfilippo syndrome is a…
Yesterday, a group of experts presented "Changing lives: Long-term outcomes of MPS IVA & VI patients" at the Satellite Symposium sponsored by BioMarin. The program began with insights on the…
Gene therapy is on the rise, and it just might be exactly what many children suffering from genetic disorders need to save their lives. Today reported that an amazing organization…
The FDA approved a human study to be conducted by Swedish Orphan Biovitrum AB to treating mucopolysaccharidosis type lllA patients with a new drug called SOB1003, reported European Pharmaceutical Review. Not only…
According to a story from EParent, the pharmaceutical company Ultragenyx released a statement confirming that the company's product MEPSEVII had received approval for public use by the Food and Drug…
REGENXBIO announced last month that the Investigational New Drug application (IND) is active for a Phase I/II clinical trial of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II)…
REGENXBIO Inc., a pharamaceutical company specializing in gene therapy, just shared that it launched a Investigational New Drug application to conduct Phase I/II trials on the new therapy they're developing,…
Recent findings from research done at the University of Pennslyvania indicate a new biomarker associated with a group of rare metabolic diseases called mucopolysaccharidoses (MPS). A biomarker is a measurable…
Losing a child is a loss like no other—the most devastating experience a parent can face. And for one daddy who lost his little girl to mucopolysaccharidosis, a very rare genetic…
As the saying goes, “Many hands make light work.” Know what’s even sweeter? When those hands are little—little tiny hands that finger paint, swing on the playground, and play monster…
Lately, I've been learning a lot about mucopolysaccharidoses (MPS). MPS covers a lot of territory. It refers to when the body is missing a particular enzyme--it doesn't matter which one:…
You may hate that these days everything seems to turn pink for the whole month of October. Or you may appreciate efforts to associate the color pink with Breast Cancer…
MPS Awareness Day has come and gone, but that won't stop us from spreading information! In honor of MPS Awareness Day (which took place on May 15th), we'd like to do…
Selfless. Alli Williams is selfless. The 17-year-old received an MPS I diagnosis as a baby. Unlike many kids with MPS, she was lucky enough to receive a bone marrow transplant--something she doesn't take lightly.…
For those unfamiliar with it, mucopolysaccharidoses (MPS) is a wicked, wicked disease. It comes in a couple of different forms, but the form one little boy lives with is MPS II,…
Say what you will about genetic testing—or testing in general—but North Carolina is finally feeling out the waters when it comes to newborn testing. The state is currently piloting a study…
Come one, come all! Well, if you have mucopolysaccharide (MPS), that is. The 30th Annual Family Conference is taking place this August in Colombus, Ohio and the National MPS Society…
The right doctor and the right hospital can make all the difference when your child is diagnosed with a rare (and frankly terrifying) medical condition. That’s what London couple Sukhi…
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