Newsletters

Message from the Executive Director  |  Family Support & Medical Conference  |  Family Advisory Working Groups  |  NNPDF Conference Scholarships  |  Conference Registration  |  Conference Accommodations  |  NNPDF Cora Sterling Endurance

Message from the Executive Director  |  Rare Disease Day  |  Community Update Series: NPC & ASMD Newborn Screening  |  Thanking Our Niemann-Pick Health Care Heroes  |  Volunteer Opportunities  |  Family

Thank You to our Supporters  |  Rare Disease Day  |  Bringing Holiday Cheer Update  |  Milestones  |  NNPDF Store  |  Family Assistance & Support Program  |  Fundraising |  ASMD Accelerate |  Family Conference

Message from our Executive Director  |  We Remember Them  |  Celebrating Milestones in 2023  |  Fundraising  |  NNPDF Action Alert! Sign on letter for the FDA in support of Arimoclomol 

Message from our Executive Director  |  Celebrating National Family Caregivers Month  |  Advocacy  |  NNPDF In Action  |  Family Assistance & Support Program  |  ASMD Feedback Interview Opportunity  |  The Assistance

Message from our Executive Director  |  Niemann-Pick Awareness Month  |  Celebrating Family Strength During Niemann-Pick Awareness Month  |  Tribute to Rhonda Kehoe  |  Quinn Madeleine Foundation  |  Milestones  |  The

Message from our Executive Director  |  Niemann-Pick Awareness Month  |  Community Connection Opportunities  |  Niemann-Pick Awareness Apparel  |  The Importance of Newborn Screening for NPD  |  Unlocking the Potential of

Message from our Executive Director  |  Family Support & Medical Conference Recap  |  NNPDF Breakthrough Award  |  NNPDF Cora Sterling Endurance Award  |  NNPDF Persevere Award  |  Lysosomal Storage Disease Advocacy

Message from our Executive Director  |  Family Support & Medical Conference  |  Conference Keynote Speaker Announcement  |  NNPDF In Action  |  Men’s Health Month  | Milestones |  Family Assistance & Support

Message from our Family Services Manager  |  Family Support & Medical Conference  |  Family Advisory Working Groups  |  Conference Accommodations  |  Conference Registration  |  Conference Keynote Speaker Announcement  |  NNPDF In

Family Support & Medical Conference  |  Early Bird Registration Discounts  |  NNPDF Cora Sterling Endurance Award  |  ASMD Scholarship Opportunity  |  Family Advisory Working Groups  |  Conference Accommodations  |  Conference Scholarships 

Message from the Board Chair  |  Family Support & Medical Conference  |  Conference Scholarships  |  Early Bird Registration Discounts  |  Family Advisory Working Groups  |  Conference Accommodations  |  NNPDF Cora Sterling

Rare Disease Day  |  Thanking our Niemann-Pick Health Care Heroes​  |  ASMD Survey Opportunity  |  Family Support & Medical Conference  |  Family Advisory Working Groups​  |  Family Conference Scholarships  |  NNPDF In

Message from the Executive Director  |  Rare Disease Week Advocacy Stipend​  |  Community Meeting with Azafaros  |  Rare & Ready Advocacy Bootcamp​  |  Courageous Parents Network Virtual Workshop​  |  Thanking

Message from the Executive Director  |  We Remember Them  |  The Benefits of Having a Great Local Care Team  |  Dangerous Blues by Stephen Policoff  |  Gift Ideas Supporting the

Niemann-Pick Awareness Month  |  NNPDF In Action  |  Celebrating #NiemannPickChampions  |  Global Niemann-Pick Disease Awareness Day  |  Bringing Holiday Cheer  |  In the Spotlight  |  ASMD Accelerate  |  Pfrieger’s Digest 

Message from the Executive Director  |  Niemann-Pick Awareness Month  |  Community Connections Opportunities  |  Family Support & Medical Conference Recordings  |  Cyclo Therapeutics Patient Newsletter  |  Orphanet Publication Alert  | 

APPROVAL! Xenpozyme for Treatment of ASMD ​  |  Community Update Series: XenpozymeMessage from the Board Chair  |  2022 Family Support & Medical Conference RecapNNPDF 2022 Awards  |  NNPDF 2022 Board

Message from the Board Chair  |  2022 Family Support & Medical ConferenceNPC Family Advisory Working Groups  |  NNPDF Community Survey  |  ASMD Community SurveyASMD Accelerate  |  Duke-Margolis Center Update  | 

2022 Family Support & Medical Conference NNPDF Cora Sterling Endurance Award  | NNPDF Research Fellowship Applications INPDA – ASMD Webinar Recording  |  ASMD Accelerate  |  Pfrieger’s Digest Transport NPC  

Message from Executive Director  |  2022 Family Support & Medical Conference Family Advisory Working Groups  |  Family Conference Scholarships Joele Ruppert & Joseph Colton ASMD Scholarship  |  NNPDF Cora Sterling

APPROVAL!  |  Message from Board Chair  |  2022 Family Support & Medical Conference Family Advisory Working Groups  |  Family Conference Scholarships NNPDF Cora Sterling Endurance Award  |  Joele Ruppert &

Message from the Executive Director  |  Rare Disease Week  |  Rare Disease DayTaking the Lead with ASMD – Sanofi Presentation  |  NNPDF In Action  |  Thank You Kara AyikHelp for

Message from the Executive Director  |  2021 RemembrancesShop AmazonSmile  |  Milestones  |  In the Spotlight  |  Invisible MannersCommunity Update Webinar Series  |  2022 Family ConferenceAllStripes NPC Research Program  |  Truly

Message from the Executive Director  |  Niemann-Pick Awareness MonthHelp for the Holidays  |  In the Spotlight  |  2022 Family ConferenceGiving Tuesday  |  Coffee & Catching Up  |  Newborn Screening UpdateCommunity

Message from the Executive Director Dear NNPDF Community, Summer’s winding down is often a period reflection. Subtle changes in the length of day, extra long sunsets, heatwaves and summer storms,

Message from the Executive Director  | Family Support & Medical ConferenceNNPDF Welcomes New Board Members  |  Thank You Missy Ward  |  New Advocacy CommitteeMembership Update  |  ASMD Scholarship Opportunity  |  Milestones  |  INPDR UpdateASMD Market Research Opportunity 

Message from the Executive Director Dear NNPDF Community, As a rare disease community, we are stronger together. Recently we have seen the impact that our collective voices and collaboration can

Message from the Vice Chair Dear NNPDF Community, On behalf of the NNPDF we would like to first and foremost thank those that participated and supported our FDA Listening Session

Message from the Board Chair NNPDF Community, I appreciate the opportunity to bring you the Spring edition of the NNPDF Board Chair update. As the world looks to rebound from

Message from the Executive Director Dear NNPDF Friends and Family, Rare Disease Day is approaching.  On February 28th, families from around the world join together as one voice. Throughout the

Message from the Executive Director Dear NNPDF Friends and Family, The first month of 2021 brought hope to some of our families yet fear and uncertainty to others in the

Message from the Executive Director Dear NNPDF Friends and Family, December brings a lot of reflection. A review of the year, it’s ups and downs, and a time to pause

Message from the Executive Director Dear NNPDF Friends and Family, Thanksgiving is almost here and while this year’s gatherings will be smaller and quieter than normal, the pause we take

Message from the Executive Director Dear NNPDF Friends and Family, October is Niemann-Pick Awareness month and throughout this month NNPDF has been raising awareness of Niemann-Pick disease through media interviews,

September is Newborn Screening Awareness Month In recognition of Newborn Screening Awareness Month we are sharing the Firefly Fund’s September Firefly Chat, which is sharing a recent discussion from the

Message from the Executive Director Dear Friends, As summer comes to an end many of our families are starting a new school year and adapting to distance learning, hybrid schedules,

NNPDF Family Support & Medical Conference Recap Thank you to the 290 participants who joined us for this year’s virtual conference weekend! Our speakers, break out session leaders, and family

Message from the Executive Director Dear Friends, The past few months presented unexpected challenges for our community.  COVID19 forced us to make changes to our daily life, learn new phrases

Message from the Executive Director Dear Friends, I’m excited to announce that the NNPDF Conference website and registration for the 2020 NNPDF Family Support and Medical Conference are OPEN! As

Message from the Executive Director Dear Friends, The month of April has been filled with challenges that we couldn’t have imagined only a few months ago. Keeping our families healthy

Message from the Executive Director Dear Friends and Families, The world has changed a great deal since our last Newsletter. The COVID19 pandemic is creating unprecedented challenges in our daily

Message from the Board Chair NNPDF Community, Recently NNPDF Executive Director, Joslyn Crowe, and I attended the annual WORLD (We’re Organizing Research on Lysosomal Diseases) scientific meeting. This 16th annual

Message from the Executive Director Dear Friends and Families, As the new year begins, we at NNPDF are working on your behalf to continue the positive community spirit and momentum