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The PSC Attends Meetings in Washington D.C.

May 2024

The Pediatric Dermatology Research Alliance (PeDRA) organized a meeting to the National Institutes of Health (NIH) and the United States Food and Drug Administration (FDA).

 

The PHACE Syndrome Community accepted an invitation to attend the meeting to speak on behalf of all families and individuals with PHACE syndrome. Moureen Wenik, PSC's Community Program Manager, along with two other patient organizations, Gorlin Syndrome and Global Parents for Eczema Research, and PeDRA Patient Advisory Chairwoman were there to represent the needs of patients. They were joined by Mike Siegel, PeDRA Executive Director, and the PeDRA Board of Directors to discuss the importance of the NIH,  and the role they play in the rare disorder space.

The group met with Lindsey A. Criswell, M.D., M.P.H., D.Sc.  Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) , and multiple other NIH institutes to discuss the burden of rare syndromes and disorders on families and patients who are living with their disorder. Discussion ranged from the need for funding for research to mental health challenges of patients, as well as the roadblock many patient's face with access to knowledgeable physicians and misdiagnoses. 

The FDA meeting was held with dermatologist Dr.Jill Lindstrom, Director, Division of Dermatology and Dentistry. This meeting included the importance of patient centered outcomes in research, and the need for FDA approval for off-label therapeutics, allowing insurance companies to cover the prescribed drug, but most importantly for patients to have ease of mind when being prescribed a known medicine to help them.  

The PeDRA Corporate Council, which was established as a step towards harmonizing goals and providing mutual benefit for all stakeholders in the pediatric dermatology space, had a meeting on Monday afternoon. The meeting was a collaborative session to determine how PeDRA can best serve physician scientists, patient groups, and and corporate partners. 

The PSC looks forward to continued collaboration with PeDRA and all the stakeholders they serve. 

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11th Annual Race for PHACE 

September 7-15, 2024
Organize an event and get your state on the map! 

 

Sign up your team and join the captain's meeting on May 28, 2024 @8:00 pm. 

 

Use this link to register your team.

Ryan is planning an event in Chicago. Learn why he is organizing a Race for PHACE. 

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Rare Caregiver Respite Program

This first-of-its-kind assistance program is designed for caregivers of a child or adult diagnosed with a rare disorder. NORD understands that caring for a loved one is a generous gift that demands significant amounts of time, attention, patience and dedication.

The Respite Program provides financial assistance to enable the caregiver a break to attend a conference, event or simply have an afternoon or evening away from caregiving. Financial assistance will be granted up to $800 annually for those who qualify.

What care may be covered?

  • Registered Nurse (RN)

  • Licensed Practical Nurse (LPN)

  • Certified Nursing Assistant (CNA)

  • Home Health Aide (HHA)

  • Non-professional caregiver

Who is eligible?

The caregiver must be a US citizen or permanent resident of the United States for at least six months with evidence of residency, and be willing to undergo a financial evaluation of need to determine if they fall within the program’s financial eligibility guidelines. Additionally, the patient for whom care is being provided must have a confirmed rare disease diagnosis.

How do you apply?

Awards are granted on a first come, first served basis to eligible individuals. There is no fee to apply. Contact a Patient Services Representative at 203.616.4328 or email CaregiverRespite@rarediseases.org.

 

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