News

June 22, 2020June 22, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will ... Read more

June 18, 2020June 18, 2020

Lysogene CEO Karen Aiach, Inspired by Daughter, Determined to Treat Sanfilippo

News

Karen Aiach never imagined she’d one day start a drug company dedicated to curing a fatal genetic disease. But that was before 2005, when the French business consultant gave ... Read more

June 11, 2020June 11, 2020

FDA Puts Hold on LYS-SAF302 Gene Therapy Trial for Sanfilippo Type A

News

The U.S. Food and Drug Administration (FDA) has issued a hold on Lysogene‘s trial of its gene therapy candidate LYS-SAF302 for Sanfilippo syndrome type A. LYS-SAF302 is a one-time ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

News

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a ... Read more

June 4, 2020June 4, 2020

Seelos Seeks Natural History Data, Rare Disease and Orphan Drug Designations for SLS-005

News

Seelos Therapeutics and the U.S. Food and Drug Administration (FDA) are continuing to discuss the use of Sanfilippo syndrome natural history data as a control for a pivotal U.S. ... Read more

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

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