According to a publication from ET Healthworld, some 21 rare disease patients in India's northern state of Uttar Pradesh are still awaiting governmental financial assistance to which they are legally…
According to a press release from the French biotechnology company Inventiva, the company has completed recruitment for a phase 2 clinical trial evaluating its experimental mucopolysaccharidosis VI drug, odiparcil. About…
According to a story from globenewswire.com, the biopharmaceutical company Denali Therapeutics, Inc. has recently announced that its experimental product candidate DNL310, which is currently being developed as a treatment for…
According to a publication from BioPortfolio, China's National Medical Products Administration (NMPA — similar to the American FDA) recently approved BioMarin's mucopolysaccharidosis type IVA drug Vimizim for use in the…
According to a story from Market Screener, the biotechnology company BioMarin Pharmaceutical Inc. recently announced that the Chinese government has approved the company's drug elosulfase alfa (marketed at Vimizim) as…
According to a publication from KYN24, the Food and Drug Administration recently approved mucopolysaccharidosis type VII (MPS VII) drug Mepsevii for marketing and administration to patients in the United States.…
A recent press release from the French biopharmaceutical company Inventiva announced that the Food and Drug Administration (FDA) had granted the company's experimental mucopolysaccharidosis VI (MPS VI) drug odiparcil rare…
The Daily News recently published an announcement by researchers at Sangamo Therapeutics showing the results of its first human trial to treat two rare genetic disorders through gene-editing technology. Gene-editing (or…
According to a story from Market Screener the biotechnology company Bioblast Pharma has announced a new partnership with Team Sanfilippo a nonprofit foundation which is committed to medical research related…
According to a story from globenewswire.com, the biopharmaceutical company Lysogene and the drug developer Sarepta Therapeutics, Inc., have recently announced that the first patient has been dosed in a Phase…
According to an article from R&D, researchers at the New York City-based Icahn School of Medicine at Mount Sinai have entered an agreement with two pharmaceutical companies to aid in…
Xconomy.com has just released that funds have been raised for a variety of companies investigating therapies for rare conditions. Unfortunately, novel therapies and inventions cost million of dollars to develop. Thankfully,…
The Jaiswal family of Lucknow, the capital of Uttar Pradesh state in northern India, knows firsthand that rare diseases are not always that. CheckOrphan has reported that three of his…
Are you interested in learning about the latest trends and drug development in the rare disease field? Then you should check out a special Journalist Team Presentation from Global Data…
Meet Ethan Ethan Faneran-Burley was diagnosed at two years old with Mucopolysaccharidosis type 6 (or MPS type 6). It's a progressive, degenerative, and ultimately fatal condition. Those with MPS type…
According to a story from Newsweek, when 25 year old Rebecca Griffiths first knew that her son Reggie, now aged two, was diagnosed with Sanfilippo syndrome, she had to face…
The 2018 MPS Symposium August 2-4, 2018 People who attend the symposium can expect to learn about that latest in MPS research as well as workshops about best practices and…
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…
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…
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…
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…
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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