The Founder and President of Cure Rare Disease discusses treatments for Duchenne Muscular Dystrophy.
All in Rare Disease
#152: Janice Berliner on Brooke's Promise
Genetic counselor and author Janice Berliner shares about the fertility and pediatric genetic counseling aspects in her novel, Brooke’s Promise.
#149 CF Series: Gunnar Esiason on Patient Advocacy
Rare disease patient leader shares about his journey with cystic fibrosis and how he utilizes his experience to advocate for the community.
#141 Stan Crooke on Ultra Rare Disease Drugs
Biotech titan Dr. Stan Crooke, who developed 20+ drugs, shares about his new n-Lorem Foundation.
#140 Patti Hall on Gigantism
Author of “Loving Large” shares her son’s rare disease diagnostic odyssey.
#115 Dr. Daria Julkowska on The European Joint Program on Rare Diseases
Celebrate Rare Disease Month with the Coordinator of the EJPRD discussing rare disease research.
#102 Seth Rotberg on Huntington Disease
Patient Advocate and Motivational Speaker shares his journey with Huntington Disease.
#98 Lydia Seiders on Aplastic Anemia
Maryland State Ambassador for NORD brings her passion for patient advocacy for the rare disease community including her daughter known as #EmmaStrong.
#96 Jim Cavan on Backpack Health
A mobile and cloud-based app that helps people with chronic, serious and rare health concerns better manage their health journeys.
#95 Kieger Family on Familial Adenomatous Polyposis
Author Laura Kieger and her son, Dr. Alexander Kieger, share their family’s courageous, century-long struggle with a rare genetic cancer syndrome.
#93 Team Telomere
A community for telomere biology disorders discusses dyskeratosis congenita.
#77 Rare Disease Day 2018
Recap of CT’s event and interviews with Angelman Syndrome and ALD patient advocates
#75 Eden Lord on My City Med
Rare Disease advocate and caregiver shares on the resources she has created.
#74 Antonio Maltese on Huntington's Disease
Patient Advocate shares his pre-symptomatically genetically diagnosis and the latest breakthrough in research
#70 Heather Z on Cowden Syndrome
Patient advocate shares about her two rare brain tumors and hereditary cancer syndrome.
#67 Daniel DeFabio on Disorder: The Rare Disease Film Festival
Daniel speaks about the films to be screened at the festival and the film he produced about his son's rare disease, Menkes Disease.
#63 Rare Disease Day 2017
NORD's event in Hartford is recapped including interviews with a patient advocate, Michelle Cotton, and a Connecticut representative, Dave W. Yaccarino.
#60 Dan Donovan on Onevoice
Co-Founder/CEO of Onevoice shares how his new community building software solution supports the rare disease community.
#52 Charcot Marie Tooth Interview
Patient advocates Bernadette Scarduzio and Allison Moore share about CMT, their documentary and the Hereditary Neuropathy Foundation.