This measure would designate the weekend of June 2, 2018, through June 3, 2018, as Batten Disease Awareness Weekend, and would call upon the President and Congress of the United States to enact legislation that provides more funding for research to be done on potential treatment and cures for rare diseases.

This measure would designate the weekend of June 2, 2018, through June 3, 2018, as Batten Disease Awareness Weekend, and would call upon the President and Congress of the United States to enact legislation that provides more funding for research to be done on potential treatment and cures for rare diseases.

This measure would designate the weekend of June 2, 2018, through June 3, 2018, as Batten Disease Awareness Weekend, and would call upon the President and Congress of the United States to enact legislation that provides more funding for research to be done on potential treatment and cures for rare diseases.

WHEREAS, Batten Disease is a family of rare genetic disorders of the nervous system that typically begins in childhood; and

WHEREAS, There are many forms of Batten Disease, and the mutations of at least 14 different genes are known to cause the disease; and

WHEREAS, Symptoms of the fatal disease include vision loss leading to blindness, personality and behavioral changes, seizures, intellectual decline, disruption and loss of speech, dementia, and other debilitating ailments; and

WHEREAS, Depending on the age of onset, Batten Disease usually leads to premature death when the patient is in his or her late teens or early twenties; and

WHEREAS, Batten Disease occurs in an estimated two to four of every 100,000 births in the United States; and

WHEREAS, Due to the many different forms of the disease, there is significant variability in the age of onset and symptom progression for each case; and

WHEREAS, On average, the process to reach the correct diagnosis for a patient with a rare disease could take up to 7.6 years, visits with numerous physicians, and multiple misdiagnoses; and

WHEREAS, There is no known cure for Batten Disease, and the lack of essential funding has hindered scientists ability to find a potential treatment or cure; and

WHEREAS, The federal government failed to pass House Resolution 971 introduced by Representative Gus Bilirakis in the 114th Congress, which represented an effort to increase the number of treatments for rare diseases, reduce the use of drugs that are not approved by the federal Food and Drug Administration (FDA), and bring down the average cost of rare disease medications; and

WHEREAS, The federal government has not done enough to provide funding for rare diseases, and 95 percent of the 7,000 rare diseases still have no FDA-approved treatment; now, therefore, be it

Resolved by the Senate and the Assembly of the State of California, jointly, That the Legislature does hereby proclaim the weekend of June 2, 2018, through June 3, 2018, as Batten Disease Awareness Weekend; and be it further

Resolved, That the Legislature calls upon the President and Congress of the United States to enact legislation that provides more funding for research to be done on potential treatment and cures for rare diseases; and be it further

Resolved, That the Secretary of the Senate transmit copies of this resolution to the President and Vice President of the United States, to the Speaker of the House of Representatives, to the Majority Leader of the Senate, and to each Senator and Representative from California in the Congress of the United States.

WHEREAS, Batten Disease is a family of rare genetic disorders of the nervous system that typically begins in childhood; and

WHEREAS, There are many forms of Batten Disease, and the mutations of at least 14 different genes are known to cause the disease; and

WHEREAS, Symptoms of the fatal disease include vision loss leading to blindness, personality and behavioral changes, seizures, intellectual decline, disruption and loss of speech, dementia, and other debilitating ailments; and

WHEREAS, Depending on the age of onset, Batten Disease usually leads to premature death when the patient is in his or her late teens or early twenties; and

WHEREAS, Batten Disease occurs in an estimated two to four of every 100,000 births in the United States; and

WHEREAS, Due to the many different forms of the disease, there is significant variability in the age of onset and symptom progression for each case; and

WHEREAS, On average, the process to reach the correct diagnosis for a patient with a rare disease could take up to 7.6 years, visits with numerous physicians, and multiple misdiagnoses; and

WHEREAS, There is no known cure for Batten Disease, and the lack of essential funding has hindered scientists ability to find a potential treatment or cure; and

WHEREAS, The federal government failed to pass House Resolution 971 introduced by Representative Gus Bilirakis in the 114th Congress, which represented an effort to increase the number of treatments for rare diseases, reduce the use of drugs that are not approved by the federal Food and Drug Administration (FDA), and bring down the average cost of rare disease medications; and

WHEREAS, The federal government has not done enough to provide funding for rare diseases, and 95 percent of the 7,000 rare diseases still have no FDA-approved treatment; now, therefore, be it

Resolved by the Senate and the Assembly of the State of California, jointly, That the Legislature does hereby proclaim the weekend of June 2, 2018, through June 3, 2018, as Batten Disease Awareness Weekend; and be it further

Resolved, That the Legislature calls upon the President and Congress of the United States to enact legislation that provides more funding for research to be done on potential treatment and cures for rare diseases; and be it further

Resolved, That the Secretary of the Senate transmit copies of this resolution to the President and Vice President of the United States, to the Speaker of the House of Representatives, to the Majority Leader of the Senate, and to each Senator and Representative from California in the Congress of the United States.

WHEREAS, Batten Disease is a family of rare genetic disorders of the nervous system that typically begins in childhood; and

WHEREAS, There are many forms of Batten Disease, and the mutations of at least 14 different genes are known to cause the disease; and

WHEREAS, Symptoms of the fatal disease include vision loss leading to blindness, personality and behavioral changes, seizures, intellectual decline, disruption and loss of speech, dementia, and other debilitating ailments; and

WHEREAS, Depending on the age of onset, Batten Disease usually leads to premature death when the patient is in his or her late teens or early twenties; and

WHEREAS, Batten Disease occurs in an estimated two to four of every 100,000 births in the United States; and

WHEREAS, Due to the many different forms of the disease, there is significant variability in the age of onset and symptom progression for each case; and

WHEREAS, On average, the process to reach the correct diagnosis for a patient with a rare disease could take up to 7.6 years, visits with numerous physicians, and multiple misdiagnoses; and

WHEREAS, There is no known cure for Batten Disease, and the lack of essential funding has hindered scientists ability to find a potential treatment or cure; and

WHEREAS, The federal government failed to pass House Resolution 971 introduced by Representative Gus Bilirakis in the 114th Congress, which represented an effort to increase the number of treatments for rare diseases, reduce the use of drugs that are not approved by the federal Food and Drug Administration (FDA), and bring down the average cost of rare disease medications; and

WHEREAS, The federal government has not done enough to provide funding for rare diseases, and 95 percent of the 7,000 rare diseases still have no FDA-approved treatment; now, therefore, be it

Resolved by the Senate and the Assembly of the State of California, jointly, That the Legislature does hereby proclaim the weekend of June 2, 2018, through June 3, 2018, as Batten Disease Awareness Weekend; and be it further

Resolved, That Legislature calls upon the President and Congress of the United States to enact legislation that provides more funding for research to be done on potential treatment and cures for rare diseases; and be it further

Resolved, That the Secretary of the Senate transmit copies of this resolution to the President and Vice President of the United States, to the Speaker of the House of Representatives, to the Majority Leader of the Senate, and to each Senator and Representative from California in the Congress of the United States.

Date of Hearing: June 19, 2018

ASSEMBLY COMMITTEE ON HEALTH

Jim Wood, Chair

SBPCA Bill Id:SJR 25

Author:(Wilk) – As Introduced Ver:April 16, 2018

SENATE VOTE: 38-0

SUBJECT: Batten Disease Awareness Weekend.

SUMMARY: Designates the weekend of June 2, 2018 and June 3, 2018 as Batten Disease Awareness Weekend and calls upon the President and Congress of the United States to enact legislation that provides more funding for research to be done on potential treatment and cures for rare diseases. Specifically, this resolution makes legislative findings and declarations related to Batten Disease, including:

1. Batten Disease is a family of rare genetic disorders of the nervous system that typically begins in childhood;
2. There are many forms of Batten Disease, and the mutations of at least 14 different genes are known to cause the disease;
3. Symptoms of the fatal disease include vision loss leading to blindness, personality and behavioral changes, seizures, intellectual decline, disruption and loss of speech, dementia, and other debilitating ailments and depending on the age of onset, Batten Disease usually leads to premature death when the patient is in his or her late teens or early twenties.
4. Batten Disease occurs in an estimated two to four of every 100,000 births in the United States and due to the many different forms of the disease, there is significant variability in the age of onset and symptom progression for each case and on average, the process to reach the correct diagnosis for a patient with a rare disease could take up to 7.6 years, visits with numerous physicians, and multiple misdiagnoses;
5. There is no known cure for Batten Disease, and the lack of essential funding has hindered scientists’ ability to find a potential treatment or cure;
6. The federal government failed to pass House Resolution 971 introduced by Representative Gus Bilirakis in the 114th Congress, which represented an effort to increase the number of treatments for rare diseases, reduce the use of drugs that are not approved by the federal Food and Drug Administration (FDA), and bring down the average cost of rare disease medications; and,
7. The federal government has not done enough to provide funding for rare diseases, and 95% of the 7,000 rare diseases still have no FDA-approved treatment.

FISCAL EFFECT: None

COMMENTS:

1. PURPOSE OF THIS RESOLUTION. According to the author, this resolution declares June 2-3, 2018 as Batten Disease Awareness Weekend to honor the individuals and families who have experienced the devastating pain that this disease causes. The author points out that this resolution brings attention to the shortfall in funding from the federal government for research on Batten Disease and other rare diseases. The author states that Sebastian Velona, the namesake for Project Sebastian, was diagnosed with Batten CLN8 at a young age. Project Sebastian has helped establish a continuously growing base of supporters, researchers, and doctors working to find a treatment or cure for Batten Disease. The author concludes that it is crucial that the Legislature call attention to organizations, such as Project Sebastian that serve to bring people together for the united purpose of actively pursuing improvements in their communities.
2. BACKGROUND. Batten Disease is an inherited disorder of the nervous system that usually appears between the ages of five and 10. Early symptoms include, vision problems and seizures and in rare cases early signs may include personality changes or clumsiness. This disease rapidly progresses leaving many individuals blind, bedridden, some cases mentally incapacitated, and is often fatal by the late teens or twenties. The National Institute of Neurological Disorders and Stroke estimate that approximately two to four individuals out of 100,000 in the U.S. are diagnosed with this rare disease. Batten disease is caused by a genetic mutation and there is currently research underway trying to understand this complex rare disease.

REGISTERED SUPPORT / OPPOSITION:

Support

None on file.

Opposition

None on file.

Analysis Prepared by: Monica Montano / HEALTH / (916) 319-2097

 THIRD READING 

Bill No: SJR 25

Author: Wilk (R), et al.

Introduced: 4/16/18

Vote: 21

SUBJECT: Batten Disease Awareness Weekend

SOURCE: Author

DIGEST:

This resolution designates the weekend of June 2, 2018, through June 3, 2018, as Batten Disease Awareness Weekend, and calls upon the President and Congress of the United States to enact legislation that provides more funding for research to be done on potential treatment and cures for rare diseases.

ANALYSIS: This resolution makes the following legislative findings:

1. Batten Disease is a family of rare genetic disorders of the nervous system that typically begins in childhood. It occurs in an estimated two to four of every 100,000 births in the United States.
2. Symptoms of the fatal disease include vision loss leading to blindness, personality and behavioral changes, seizures, intellectual decline, disruption and loss of speech, dementia, and other debilitating ailments.
3. Depending on the age of onset, Batten Disease usually leads to premature death when the patient is in his or her late teens or early twenties.
4. On average, the process to reach the correct diagnosis for a patient with a rare disease could take up to 7.6 years, visits with numerous physicians, and multiple misdiagnoses.
5. There is no known cure for Batten Disease, and the lack of essential funding has hindered scientists’ ability to find a potential treatment or cure.
6. The federal government has not done enough to provide funding for rare diseases, and 95% of the 7,000 rare diseases still have no federal Food and Drug Administration-approved treatment.

This resolution proclaims the weekend of June 2, 2018, through June 3, 2018, as Batten Disease Awareness Weekend, and calls upon the President and Congress of the United States to enact legislation that provides more funding for research to be done on potential treatment and cures for rare diseases.

FISCAL EFFECT: Appropriation: No Fiscal Com.: No Local: No

SUPPORT: (Verified 4/23/18)

None received

OPPOSITION: (Verified 4/23/18)

None received

Prepared by: Melissa Ward / SFA / (916) 651-1520

4/25/18 14:49:18

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