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• Healthcare Legislation

• Local Introductory Meeting

• MisGivings Fundraiser

• Wine for a Cause Fundraiser

• Festival of Genomics

The rare disease community has many supporters active in legislative efforts through State Representatives, Senators, Congressmen/women, Advocacy organizations, Hospital administrations, Patients and Caregivers, Healthcare workers, Healthcare industry, and more. They initiate bills and promote action steps as bills work through Committees with the goal of passing votes through the House and Senate to become Laws.

Although we are fortunate to have those working for us to build a better health care system, they can not do their job alone; for most positive outcomes, our help is needed to spread awareness of the plight of the rare community and the need to pass legislation as swiftly as possible. (For current rare disease policy issues and Action Alerts, scroll down to learn more about National Organization for Rare Disorders and Rare Disease Legislative Advocates and these organization's efforts to assist the rare disease community).

There is a lot we can do to aid legislative efforts and we can begin with acts as simple as making telephone calls and/or sending emails to our own State Representatives and Senators to explain what it is like to live or work with rare disease and ask for their support for bills in play. EVERY call/email counts!

In words of a legislative aide who worked with Congressman Joseph Kennedy, "When we receive a call about an issue we take note. When we begin to receive multiple calls on an issue, we realize we have a problem to solve". YOUR call/email could be the one that makes the difference! Don't hold back; make that call today.

Senate Bill 2984

 Media

 At the beginning of 2019, RNE geared up to introduce ourselves to the local community as our 3rd year anniversary approached at the end of April. We invited local community stakeholders, including the local hospital, visiting nurses, YMCA, local banking institutions, the media, and more, to hear about rare diseases and RNE's programs aiming to make a positive difference. We also invited industry to learn about our efforts.

Thank you to everyone who attended and learned more about the rare disease community and RNE's efforts to serve this community.

To watch a recording of this event, click here.

Speakers:

• Julie Gortze, RNE Founder

• Lisa Deck, Rare Disease Advocate and Patient

• Mark Korson MD, Clinical Biochemical Geneticist

• John Campbell, Clinical Development Director, Pharma

• Josephy Kennedy III, Congressman

• ​Joseph McKenna, MA State Representative

• Paul Feeney, MA Senator

Congressman Joe Kennedy III Transcript

May 3rd, 2019

"I've talked to many families that have said, 'the only thing worse than knowing that there's no cure for my child, is knowing that there is, but I can't afford it.' That cannot be acceptable today in this country." (Congressman Kennedy III)

Sharing Congressman Kennedy's powerful speech. Thank you, Congressman Kennedy, for illuminating such a vital and worthwhile organization. (BC Mosher)

Congressman Joe Kennedy III: "The challenges that so many families across our Commonwealth, across our country, face for those that are struggling, have a family member struggling with a rare disease it is things that I've come to know and appreciate and to learn is this is not an issue that affects just a single patient, this affects the entire family. It affects the family in terms of the ability of their loved ones to provide the care that is necessary. It affects the family, clearly, in terms of the financial burden that oftentimes comes down on family members.

And without the robust federal understanding, support, structures, incentives, and protections put in place, it's one that all too often ends up with family members having to choose between how you care for one loved one versus another one.

And that's not the burden that the wealthiest, most successful nation on the planet should be putting on families because somebody is sick.

We, my responsibility, I think all of us in Congress, need to try to find a way:

1 - To ensure the proper structures and incentives to make sure we continue to innovate so that we find cures for these conditions and these diseases. And that's not just mitigating the symptoms, but actually curing the underlying illness.

That we recognize then that as those cures are developed, that the time, the complexity, the costs associated with them, that that does not lead to cost structures that then would put those medicines out of reach for so many families.

I've talked to many families that have said, 'the only thing worse than knowing that there is no cure for my child, is knowing that there is, but I can't afford it.'" That cannot be acceptable today in this country.

2- Is making sure that for the millions of Americans that are unaware about how this system operates - the burden that it puts on those families -- the ineffectiveness of some of the structures and incentives put in place, that we do a better job of educating them and continuing to push Congress to make the adjustments that are necessary to spread that innovation, but then to ensure that every single patient has the access to the care that they need when they need it at a price that they can afford.

Your efforts and your advocacy, the ways in which you have elevated these issues and the circumstances that - how they affect your families and families of those that you care about and families of those that you represent, have been as critically important to me as I have learned about these issues.

And as Congress does, and I do believe we will, finally start to wrestle with issues around the cost of the prescription drugs; and that we make sure we get this right because we have a duty and an obligation, as I said:

1 - To make sure that people -- everybody across this country gets access to medication that they need when they need it, and at a price they can afford. But

2- that we ensure that innovation continues so that we can continue to make progress on these conditions.

We have a chance in Congress to actually restructure this field. And I will be looking to you to make sure that as we do so, that we keep the focus on the patients and their families and to make sure that we do this right".

*Transcript Credit to BC Mosher

 RNE's first event fundraiser was held on January 20th, 2018 and included dinner, a comedy show by Dave Kane, radio talk show host and comedian, and silent auction.

Dave has been involved in the field of radio for over 40 years; he worked as a producer for WJAR radio and filled in for the other hosts. In 1981, Dave joined WARA in Attleboro, Massachusetts and eventually became station manager. From that point forward, Dave carved a successful niche in Rhode Island talk radio, spending time at various stations throughout the region, including WPRO and WHJJ, two of the largest and most powerful talk stations in the state. Since January of 2014, Dave has been a weekend host on WARA Saturday mornings. His show Kane and Company can currently be heard in the 9am-12pm time slot.

Dave's fundraising and charity efforts include Misgivings, and Just for Funds (a concept in which the venue and entertainment are provided for fundraising organizations). Dave also created Christmas is For Kids. Each year, this charity provides gifts to an average of 800 to 1,000 children in need each Christmas. This program started in 1983 and is supported by the Greater Attleboro Area Council for Children.

City Spirits partnered with RNE for our "Wine for a Cause" fundraiser throughout 2018. For the month of June, City Spirits donated 100% of the profits from Fitvine wines to Rare New England. Thank you City Spirit for the support and successful fundraiser!

Donations helped support:

• Annual patient/family conferences

• Annual Rare Disease Day events

• Teen events and programs

• Support programs

• And more…

 Festival of Genomics and Rare New England partnered to offer a stage area targeted to patients and families living with rare diseases!

Front Line Genomics has a mission to help deliver the benefits of genomics faster. Through their website, magazine and the Festivals of Genomics, they support scientists, clinicians, business/research leaders and officials, from academia, research institutes, industry, healthcare and government organisations to realise the true potential of genomic medicine.
Each year they host Festivals in 3 locations - London, San Diego and Boston "to bring the benefits of genomics to patients, faster".

Festival of Genomics offers an abundance of information about genomics and brings together researchers, healthcare organizations, academia, patient advocacy organizations, industry, and more! These stakeholders who are living and working within the field of genomics have an opportunity to share insight and learn new ways to incorporate this knowledge to benefit patients.