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May 22, 2020

TOPIC: Featured News, Patients & Members, Advocacy, Head of the Herd

Head of the Herd: Emile Najm, KAT6A Foundation

Posted at May 5, 2020 08:46 am by Laura Mullen

At the head of the herd is… Emile Najm, Chief Executive Officer, KAT6A Foundation

Emile most frequently checks his…  Facebook account

Emile advocates for those living with… A rare syndrome resulting from a mutation in the KAT6A gene.

Emile is located in… West Nyack, New York

How Emile got here… Emile Najm’s son was diagnosed with KAT6A syndrome in 2017. Since then,… Read More

May 13, 2020

TOPIC: Featured News, Patients & Members, Research, Advocacy

NORD to Host an Externally-led Patient-Focused Drug Development Virtual Meeting on Krabbe Disease, Thursday, October 29

Posted at May 5, 2020 02:06 pm by Laura Mullen

NORD, KrabbeConnect and Partners for Krabbe Research invite you to register for an externally-led Patient-Focused Drug Development (EL-PFDD) virtual meeting on Krabbe disease, taking place Thursday, October 29, 12:30-4:30pm ET. Patients and caregivers impacted by Krabbe disease who are interested in participating at the virtual meeting as panelists should complete this Read More

April 28, 2020

TOPIC: Featured News, Patients & Members, Advocacy

Historic “Voice of the Patient” Report on Pyruvate Kinase Deficiency Now Available on FDA Site

Posted at April 4, 2020 08:59 am by Laura Mullen

“…As we pull into the hospital, she starts screaming that she doesn’t want to be there, and she wants to be home… No matter how much her doctors want to make her feel better, and she’s also three, so it’s hard to explain to her that this will make you feel better. All she knows is I’m… Read More

April 24, 2020

TOPIC: Featured News, Advocacy, COVID-19

NORD Joins 400+ Groups to Prevent Discrimination During COVID-19 Triaging

Posted at April 4, 2020 09:00 am by Laura Mullen

Between 25 and 30 million Americans have a rare disease, many of whom are disproportionately at risk of getting sick from COVID-19. According to the Centers for Disease Control and Prevention, in addition to older adults, people of any age who have serious underlying medical conditions like chronic lung disease or asthma, serious heart conditions,… Read More

April 20, 2020

TOPIC: Featured News, Advocacy, COVID-19

Update on NORD’s Recent Advocacy Work on COVID-19 for the Rare Community

Posted at April 4, 2020 11:15 am by Laura Mullen

In this time of crisis and uncertainty, NORD’s policy team has been working to pursue policies at the federal and state levels to address COVID-19 related concerns critical to the rare disease community. 

Protecting Access to Necessary Medical Treatment During the Pandemic

Social distancing is critical for slowing the spread of the coronavirus. But, for many rare… Read More