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May 28, 2020May 28, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

News, syndicated

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene ... Read more

May 28, 2020May 28, 2020

Cure Sanfilippo Grants $380,000 to Type C Gene Therapy Development

News

Cure Sanfilippo Foundation has granted $380,000 to the New York-based biotechnology company Phoenix Nest to support the development of the first-ever gene therapy to treat children with the rare ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

News, syndicated

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session ... Read more

May 21, 2020May 21, 2020

SLS-005 Granted FDA’s Rare Pediatric Disease Designation for Sanfilippo

News

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to Seelos Therapeutics‘ SLS-005 (trehalose) to treat Sanfilippo syndrome, the company has announced. The designation is granted to product applications for ... Read more

May 21, 2020May 21, 2020

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

cystic fibrosis, News, syndicated

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s ... Read more

May 20, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

News, syndicated

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. ... Read more

May 18, 2020May 18, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

News, syndicated

May 14, 2020May 14, 2020

RDMD Secures $14M in Financing to Expand Platform for Sanfilippo, Other Rare Disorders

News

Healthcare technology company RDMD has raised $14 million to further expand its platform seeking to accelerate research and treatment development for rare diseases, including Sanfilippo syndrome. The Series A ... Read more

May 11, 2020May 11, 2020

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

News, syndicated

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. ... Read more

May 7, 2020May 7, 2020

FDA Grants Orphan Drug Status to SLS-005 for Treating Sanfilippo

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Seelos Therapeutics‘ SLS-005 (trehalose) to treat Sanfilippo syndrome, the company has announced. Orphan drug designation aims to encourage the ... Read more

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