Written by Dawn Laney, MS, CDC, CCRC The journey to a Fabry disease diagnosis is rarely a clear, straight path. Fabry can mimic more common health issues and lead patients…
In an exciting press release, gene therapy platform company Lysogene announced that it had received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) and Research Ethics Committee. As…
According to a recent press release from the Massachusetts-based biotechnology company Avrobio, Inc., the first patient has been dosed in the Company's phase I/II clinical study of their investigational cystinosis…
According to a recent publication from Moneycontrol, patient advocacy groups in India are calling for the country's Minister of Health and Family Welfare, Harsh Vardhan, to establish a provisional system…
According to a press release from American biotechnology company Neurogene, the Company has kicked off a sponsorship of Invitae Corporation's "Detect Lysosomal Storage Diseases" program, which will offer genetic testing…
The Heritage Florida Jewish News reported a story about one sister's journey to her Gaucher Disease diagnosis, thanks to her older sister's wedding prep. What is Gaucher Disease? Gaucher disease…
I had the chance to chat with Amber Olsen, the founder of The United MSD Foundation, and mother to Willow. This is their story. Willow had been a seemingly healthy…
In an effort to better understand patient experiences, researchers at the Massachusetts General Hospital are surveying people with lysosomal storage diseases about how they view their treatments, reports CheckOrphan. The…
Irish drugmaker, Shire, announced earlier this month it will bring new genetic disorder drugs to India, stepping up its worldwide presence. Shire is unique in that it's one of the…
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