Posts by: Hawken Miller

News, syndicated

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how ... Read more

November 9, 2020November 10, 2020

UK Mom Knows Fight Far From Over After Losing Baby to Gaucher Type 2

News

Note: This story was updated Nov. 10, 2020, to clarify that Foggin volunteers with the International Gaucher Alliance and does not hold a formal position with Prevail. In addition, the ... Read more

September 28, 2020September 28, 2020

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

News, syndicated

It took one year for Dona Krystosek to get a diagnosis for her son, Levi, after he was born. The family received three misdiagnoses of fatal diseases until they found ... Read more

August 12, 2020August 12, 2020

New Streaming Channel Showcases Rare Disease Films

News, syndicated

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films ... 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 son, ... Read more

March 26, 2020March 26, 2020

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

News

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The ... Read more

February 27, 2020February 27, 2020

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

News

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the complex process in its Feb. 20 ... Read more

February 12, 2020February 12, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

News

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, ... Read more

February 6, 2020February 6, 2020

IGA, Kantar Partner to Build Global Gaucher Registry for Type 2 and 3 Patients

News

The International Gaucher Alliance (IGA), which includes members from 54 countries, has announced a partnership with the London-based data consulting company Kantar to manage its new patient registry for neuronopathic Gaucher disease, ... Read more