Brain pops are available, order yours today! You could even hold on to them and hand them out on Halloween as a tasty awareness treat! ORDER BRAIN POPS Looking just to raise funds for the mission? Join our Team Fundraising goal! TEAM FUNDRAISING
Each year Global Genes convenes one of the world’s largest gatherings of rare disease patients, caregivers, advocates, healthcare professionals, researchers, partners and allies. Due to the COVID-19 global pandemic, and the threat this poses to the health and safety of our rare disease community, we are replacing this highly-anticipated event with the Global Genes LIVE! …
Global Genes LIVE! A RARE Patient Advocacy (un)Summit Read More »
United Leukodystrophy Foundation and the CTX Alliance Invite You to a Virtual CTX Community Forum. 2:00-3:00 PM (Eastern US Time) The Unlock CTX PFDD meeting held September 14, 2021 was a landmark event for the CTX community. Thanks to all who participated -- especially those who shared candid portraits of what it is like to …
Children experience their world through play, creativity, and imagination. It is how they learn and process, and provides mastery over their environment. Play becomes all the more important when faced with adversity or trauma. In a medical setting, expressive therapies can be used to help children understand and process their illness and can even have …
Cure GM1 is extremely proud to present a virtual conference for the GM1 community and families, biotech professionals, academic researchers, and physicians. Cure GM1 is the only 501(c)(3) entirely dedicated to GM1 gangliosidosis patient advocacy and GM1 research. Our virtual conference will be a series of webinars throughout the month of September. https://registration.socio.events/e/gm1
Join us for a variety of interactive and educational events, meet-ups, workshops and networking opportunities. Here you’ll have the opportunity to connect and engage with others in the rare disease community, while experiencing the sense of community and belonging we’ve missed so much. Gain insights about the latest in rare disease innovations, best practices for …
Global Genes LIVE! A RARE Patient Advocacy (un)Summit Read More »
Caregiving Spouses Led by Margaret Halter (AMN) and Roger Locy (AMN) An informal chat for the caregiving spouses of people affected by leukodystrophy. It will be moderated, but the discussion is open to anyone. Please note that this discussion will NOT BE RECORDED. You are free to participate at your comfort level, you will not …
Siblings of Leukodystrophy Led by Margaret McIntyre (VWM) An informal chat for the siblings of people affected by leukodystrophy. It will be moderated, but the discussion is open to anyone. Please note that this discussion will NOT BE RECORDED. You are free to participate at your comfort level, you will not be forced to answer …
The ULF is launching a monthly webinar series, and we need to hear from YOU! What topics would you like to learn more about? How can the ULF be more helpful to your family? What kind of resources are you in need of now? Or when you were newly diagnosed? We will take your suggestions, …
Your experience begins with our fabulous Cocktail Hour. You and your Guest(s) will be treated to amazing Action-Appetizer Stations featuring an all-star lineup of very talented local Chef’s. You will have the opportunity to peruse the awesome Silent Auction Tables as well as participate in our fantastic Raffles. https://bethanyshope.org/event/the-dinner/
Cure GM1 is extremely proud to present a virtual conference for the GM1 community and families, biotech professionals, academic researchers, and physicians. Cure GM1 is the only 501(c)(3) entirely dedicated to GM1 gangliosidosis patient advocacy and GM1 research. Our virtual conference will be a series of webinars throughout the month of September. https://registration.socio.events/e/gm1
Sister's hope Foundation presents - ALSP Community Support Group Meeting with Tanya Bardakjian, Genetics Counselor at the University of Pennsylvania Neurology Department * The Role of a Genetics Counselor as Part of the Treatment Team https://sistershopefoundation.com/events/caregiver-support-group-meeting-4/
Educational webinars on RARE neurological, neuromuscular and movement disorders jointly organized by the European Reference Networks for Rare Neurological Diseases (ERN-RND) and Neuromuscular Diseases (ERN EuroNMD) and the European Academy of Neurology (EAN). The speaker is an associate professor of genetics at the University Pierre and Marie Curie (UPMC), Paris, France. Dr Mochel leads the …
#PauseforTheGFPD is our worldwide awareness day. Every year, on October 5th, the Global Foundation for Peroxisomal Disorders pauses to recognize and honor all of the families that have lost a child or are currently fighting a peroxisomal disorder. Show support by taking a moment to pause for individuals & families devastated by this disorder. Even …
Educational webinars on RARE neurological, neuromuscular and movement disorders jointly organized by the European Reference Networks for Rare Neurological Diseases (ERN-RND) and Neuromuscular Diseases (ERN EuroNMD) and the European Academy of Neurology (EAN). The speaker is Child Neurologist and researcher at the Hospital Bicêtre and the Institute of Brain and Spine in Paris, France. Her …
X-linked adrenoleukodystrophies – Update by Caroline Sevin Read More »
Each year, the NORD Summit, one of the largest multi-stakeholder events in rare disease, brings together participants spanning the rare community, including rare disease experts and leaders from patient advocacy groups, government, industry, and academia to discuss the most current and critical topics related to rare diseases and orphan products. https://nordsummit.org/
Global DARE Foundation is hosting a webinar presented by Professor Dr. Jean-Marc Nuoffer, MD from the University Hospital in Bern, Switzerland will discuss Phytanic Acid Analysis and Interpretation https://www.eventbrite.com/e/phytanic-acid-analysis-and-interpretation-lab-clinical-considerations-tickets-166358481697
Despite an increase in specialized pediatric palliative care programs there remain inadequacies in end of life (EOL) care for children. This presentation will review the perspectives of 28 Spanish- and English-speaking bereaved parents who detailed qualitative experiences about their children’s end of life care. Parents’ perspectives from the study highlight the importance of 1) supporting …
2021 Pediatric Palliative Care WebinarsBereaved Parents View on End-of-Life Care Read More »
Educational webinars on rare neurological, neuromuscular and movement disorders jointly organized by the European Reference Networks for Rare Neurological Diseases (ERN-RND) and Neuromuscular Diseases (ERN-EuroNMD) and the European Academy of Neurology (EAN). The speaker is consultant child neurologist and associate professor at the Emma Children’s Hospital, Amsterdam University Medical Centres, the Netherlands.She is an expert …
ERN-RND – Pelizaeus-Merzbacher disease and related disorders’ by Nicole Wolf Read More »
Sister's hope Foundation presents - ALSP Community Support Group Meeting https://sistershopefoundation.com/events/caregiver-support-group-meeting-5/
NTSAD’s goal is to provide opportunities for members of our NTSAD Community living near and far to engage, connect, bid Sue farewell, and above all else — support families. NTSAD relies on industry members, researchers, and rare allies to invest in our families’ futures through partnership, philanthropy, and support of our events. Consider sponsoring the …
NTSAD’s goal is to provide opportunities for members of our NTSAD Community living near and far to engage, connect, bid Sue farewell, and above all else — support families. NTSAD relies on industry members, researchers, and rare allies to invest in our families’ futures through partnership, philanthropy, and support of our events. Consider sponsoring the …
ALD Connect Virtual 2021 Annual Meeting and Patient Learning Academy on November 12 and 13, 2021!https://aldconnect.org/calendar/ald-connect-2021-annual-meeting-and-patient-learning-academy-virtual/
With the growing emphasis on value-based payments in healthcare, there is significant potential for enhanced access to pediatric palliative care services. This discussion will highlight the challenges to access of PPC services in traditional reimbursement models, and it will discuss existing state models that have been developed. Finally, a process for exploring policy and legislative …
Alex TLC Community Weekend on the 20th & 21st November, is designed to bring together sufferers of leukodystrophy and their families, alongside doctors, researchers and scientists from around the world to meet, learn and support one another over a weekend of talks and activities. Dr John Livingston will be joining to give his update about …
