Inheritable Disorders Grants

Clinical Research Awards for Neurofibromatosis: Request for Letters of Intent

The Children’s Tumor Foundation announces a call for Letters of Intent for Clinical Research Awards of up to $100,000. Clinical Research Awards seeks broad-thinking novel ideas to conduct pilot clinical trials of candidate therapeutics for the treatment of tumors and other manifestations of NF1, NF2 and schwannomatosis; OR innovative studies ancillary or adjunct to clinical trials that - if successful - will contribute to the advancement of effective clinical therapies for neurofibromatosis.

Examples of projects encouraged may include but are not limited to:
- Biomarker studies adjunct to an ongoing clinical trial (may be e.g. molecular, imaging or genetic biomarkers, or other novel outcome parameters) that may better inform on and ultimately help accelerate a clinical trial.
- Preclinical/clinical collaborative studies (‘clinical co-trials’). Note however that all studies must include a clinical element – preclinical-only studies are not eligible.
- Applications to fund pilot neurofibromatosis clinical trials are welcomed an encouraged.

It is anticipated that CTF will fund up to four Clinical Research Awards of up to $100,000 (including overheads/indirect costs).

Application Process and Timeline
LOI’s due: September 15, 2010
Applicants notified: October 4, 2010

Successful LOIs will be invited to submit a full application.

Full applications due: November 15, 2010
Applicants notified: Mid-December 2010
Funding commences as early as: January 2011

Before submitting your LOI, you are strongly encouraged to contact me to discuss your idea.

Kim Hunter-Schaedle, Ph.D.
Chief Scientific Officer
Children's Tumor Foundation
Khunter-schaedle@ctf.org
Tel: 212-344-6633 x231

Alpha-1 Foundation Grant Opportunities

LETTER OF INTENT INFORMATION

Deadline for our receipt of your in-cycle LOI, Biosketch and Keyword List: September 21, 2010

The first step in the process of a grant application to the Alpha-1 Foundation is to submit an LOI, Biographical Sketch for the LOI and LOI Keyword list. All three documents must be submitted to the Foundation by September 21, 2010 in order to be invited to submit a full grant application. The submission of an LOI is required for each grant category. An applicant must submit an LOI in order to submit a grant application. A grant application that is submitted without an LOI will result in an automatic denial of funding and the application will be returned to the investigator.

For information about the Foundation’s in-cycle grant opportunities, please contact David Fernandez, Grants Coordinator, at dfernandez@alpha-1foundation.org or at 305-567-9888 ext 242.

GRANT APPLICATION INFORMATION

Deadline for our receipt of your in-cycle grant application: February 18, 2011

If your LOI is approved, the Alpha-1 Foundation will invite you to submit a full grant application. Please note that a LOI must be submitted, reviewed and approved prior to submitting a grant application.

For information about the Alpha-1 Foundation’s in-cycle grant application process or requirements, please contact Randel Plant, Director of Research Administration, at rplant@alpha-1foundation.org or at 305-567-9888 ext 252.

IN CYCLE RESEARCH GRANT CATEGORIES:

Bridge Grant

Funding Level: Maximum of $25,000 may be requested.

Period: Up to 1 year of support is available.

The objective of this grant is to provide support for excellent Alpha-1 Antitrypsin (AAT) Deficiency-related research projects that have been submitted to and approved by the National Institutes of Health (NIH), but cannot be supported by the available NIH funds. Thus, this award complements the granting mechanism of the NIH for projects of potential interest to individuals served by the Alpha-1 Foundation. Funds provided by the Alpha-1 Foundation through this program are intended to lead to the development of sufficient preliminary data to make AAT-related applications highly competitive in the NIH review process. Information derived from Alpha-1 Foundation supported research studies is intended to lead to solicitation of additional, subsequent funds from other agencies such as the NIH.

Ethical, Legal and Social Issues (ELSI) Related to AAT Deficiency

Funding Levels: ELSI projects may be submitted under the following grant categories: Pilot & Feasibility, Postdoctoral Research Fellowship or Research. Please see the funding levels stated above for each respective grant category.

Period: Up to 2 years of support is available, depending on the grant category in which the grant is submitted. Please see the periods stated above for each respective grant category.

The objective of this grant is to provide funds to encourage the development of new information that contributes to the understanding of bioethical, legal, economic and/or social issues associated with AAT Deficiency. Proposals may come from a broad spectrum of disciplines, including humanities, social and natural sciences, and health professions. In particular, this grant mechanism seeks to support the development of novel approaches relating to informed consent, conflicts of interest, organ allocation, genetic testing and/or genetic discrimination; however, applications may address all of the following or other related areas:

1. Surveying attitudes about genetic testing and screening; changing physician behavior and standards of care regarding testing; confidential testing; genetic counseling; public awareness and education about Alpha-1.
2. Improving the quality of life of alpha-1 antitrypsin deficient patients and their families; chronic illness, terminal illness, advance CARE planning, and end of life issues; employment issues; family planning issues; investigations of the social or economic impacts of a diagnosis of AAT Deficiency including stigma post-diagnosis.
3. The provision of an informed consent associated with AAT Deficiency testing, medical treatments, participation in research, or transplantation; other issues relating to participation in clinical trials or research studies.
4. Issues relating to genetic discrimination including access to insurance coverage, social services, medical care, employment, and/or military service; the protection of confidentiality of patients and their families; legislation relating to genetic disease and alpha-1 antitrypsin deficient individuals.
5. Equitable distribution of available medical therapies; availability and allocation of organs for transplantation associated with AAT Deficiency.
6. Intellectual property rights; ownership of tissue, organs and DNA associated with research studies and clinical trials; patents relating to genetic research.

All proposals must be hypothesis-generating or hypothesis-testing, or generate new information on conceptual topics relevant to the Foundation’s research mission. Applicants may submit this application as a Pilot and Feasibility Grant, Postdoctoral Research Fellowship Grant or Research Grant. Projects submitted for Research Grant funding must provide sufficient preliminary data to justify the Foundation’s support.

Pilot and Feasibility Grant

Funding Level: Maximum of $40,000 may be requested.

Period: Up to 1 year of support is available.

The objective of this grant is to provide funds to encourage the development and testing of new hypotheses and/or new methods in research areas relevant to AAT Deficiency. Proposed work must be hypothesis generating or hypothesis testing, reflecting innovative approaches to important questions in AAT research or development of novel methods, and providing sufficient preliminary data to justify the Foundation’s support. Results from Pilot and Feasibility Grants should lead to the submission of applications for funding from other agencies (i.e. NIH). At the conclusion of the funding term, applicants are expected to apply for further funding by other mechanisms or from outside agencies. The award is not intended to support continuation of programs begun under other granting mechanisms.

Postdoctoral Research Fellowship Grant

Funding Level: Maximum of $35,000 per year may be requested.

Period: Up to 2 years of support is available.

The objective of this grant is to provide support for postdoctoral research fellows who are starting their research careers and are working in the laboratories of established researchers or conducting research with the mentorship of established researchers. In addition, this grant category is intended to provide support for postdoctoral research fellows who intend to purse a career in AAT research. Applications will be accepted from candidates holding an M.D., Ph.D. or equivalent degrees who are interested in conducting basic science, clinical research, or ethic, legal, social issues studies related to AAT Deficiency. Applicants must indicate a commitment to AAT-related research by focusing 50% of their time to AAT Deficiency research or clinical practice.

Research Grant

Funding Level: Maximum of $100,000 per year may be requested.

Period: Up to 2 years of support is available.

The objective of this grant is to provide funds to encourage the development of new information that contributes to the understanding of the basic biology of AAT expression, and the pathogenesis/management of AAT Deficiency. In addition, consideration will be given to those projects that provide insight into the development of information that may contribute to new therapies for AAT Deficiency. All proposals must be hypothesis generating or hypothesis testing and provide sufficient preliminary data to justify the Alpha-1 Foundation’s support.

Scientific Meeting Sponsorship Grant

Funding Level: Grants are generally funded at the $5,000 level. The funds are restricted for use during a specified period of time.

Period: Varies according to request.

The objective of this grant is to provide support for scientific meetings, workshops or conferences relevant to AAT Deficiency and the Alpha-1 Foundation’s mission statement.

Travel Grant
There are 2 types of Travel Grants:

Funding Level: Grants are generally funded at a level of $1,000. The funds are restricted for use during a specified period of time.

Period: Varies according to request.

1. The Alpha-1 Foundation provides funds to support travel to attend a scientific conference or meeting to present AAT-related abstracts or posters at national and international meetings. Candidates must have an M.D., Ph.D. or equivalent degree, and at least one year of recent research experience related to AAT Deficiency. The award is intended primarily for travel support for an award recipient. If the grant is to be used for specialized training, a letter of support for the individual must be presented, and clearly indicate the visitor’s role, time commitment and objectives for the proposed training as well as institutional commitment.

1. The Alpha-1 Foundation provides funds to support cross training at an established Alpha-1 laboratory by an established Alpha-1 investigator. Training may include training on diagnostic and therapeutic procedures, research related techniques, training on specialized equipment or software applicable to AAT-related research.

Acid Maltase Deficiency Association 2010 Research Grant Opportunity

The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by a private fundraiser aimed at promoting research into Pompe Disease.

This research grant is for a one-time grant of $50,000.

The application deadline is: Friday, September 3, 2010.

Shwachman Diamond Syndrome Foundation Research Grant

Shwachman-Diamond Syndrome (SDS) is a multi-system autosomal-recessive disorder characterized by exocrine pancreatic dysfunction, and varying degrees of marrow dysfunction with cytopenias. Many associated clinical problems have also been described, including skeletal anomalies, metaphyseal dysostosis, rib abnormalities, cleft palate, dental abnormalities and others. Shwachman-Diamond Syndrome Foundation (SDSF) is a nonprofit support organization actively helping the victims of this disease. SDSF is accepting grant requests for research relevant to this disease. Any high quality, innovative research proposal deemed relevant to SDS will be considered. The following are areas of particular interest:

Investigate or evaluate potential therapies or therapeutic strategies
Evaluate predictors of bone marrow abnormalities to determine their correlation with the disease
Determine the causes of bone marrow failure and leukemia
Further delineate the pathogenesis of pancreatic disease
Determine if differences in disease complications occur dependent on persistent vs. cylic neutropenia
Investigations related to adult patient presentation, management, and care including transition from pediatric-centered to adult care

Eligibility
Persons applying for these grants must be in a faculty position with authority to hold an independent research grant (not a training position).

Letter of Intent
The potential applicant should write a one-page letter of intent to the SDSF, indicating the applicant’s position and affiliation, describing their planned project, and explaining its relevance to Shwachman-Diamond Syndrome. These letters are reviewed by SDSF’s Medical and Scientific Advisory Board (MSAB) grant review process.

Purpose
This letter will be used to identify applicants who will be asked to provide a full application.

Dates
The letter is due no later than March 12th. All those who have submitted a letter of intent will receive a reply by April 5th. All Grant Applications must be submitted by May 15th. The final grant decision will be announced by July 31st with a November 1st start date.

Terms of Support
Support may be provided for up to two (2) years in an amount not to exceed $65,000 per year. The second year funding will not be released until a Progress Report has been reviewed and approved by the MSAB. The date for the Progress Report is October 1st. Applicants may submit only one grant application at a time, and may hold only one grant at a time.

If you would like us to send you a grant application, have any questions regarding the grant process or have an interest in the disease, please contact SDSF at the address indicated on our home page, call toll free 1-888-825-SDSF (7373) or via email at info@shwachman-diamond.org.

Shwachman-Diamond Syndrome Foundation
127 Western Avenue
Sherborn, MA 01770
U.S.A.

By Phone Toll Free 888-825-SDSF (7373)
Fax: Same

By Email info@shwachman-diamond.org

Audi Gerstein Platelet Disorder Scholarship for Undergraduate/Graduate Students with Platelet Disorders

Awards are restricted to senior high school students, current college students, or adults interested in
continuing education who have ITP or a similar inherited or non-inherited diagnosed platelet disorder.

The Scholarship award is intended to assist full-time undergraduate or graduate students entering or
attending an accredited technical school, two or four year college, university or graduate school in the
United States.

The Applicant must be a U.S. Citizen. There is no GPA requirement, however, the
applicant must have acceptance to the college of their choice. Former Scholarship recipients are not
permitted to reapply (those who have won book awards are eligible). Family members of ITP patients
or PDSA board members or Scholarship committee members are not eligible to apply.

The Process
A 700 – 1,000 word essay is required which details how your platelet disorder has resulted in your
growth as a person; what you learned about your own strengths and weaknesses. If you feel its
changed you, whether emotionally or physically, describe how it changed your approach to life and
relationships. Describe how having an illness might have affected your life's s accomplishments and
helped you set goals. If you feel that having a chronic illness has helped you choose a career path,
describe how, and explain why you feel this way and what you have done to meet these educational
goals. Explain what your plans are for the future in meeting your educational responsibilities, in light of
your struggles with a platelet disorder.

An Application will be due and submitted to the PDSA no earlier than March 15 and no later than
April 15. Awards will be announced by May 15. Incomplete or late Applications will not be considered.

Scholarship award winners will be announced on our website. The essays and applications will be
reviewed and judged by an impartial panel selected by the PDSA Board of Directors.

Scholarship Funds
Scholarship Funds will be awarded in a one-time payment of $1,000 during the academic year.
Payments will be made directly to the institution prior to the start of the fall semester.

Platelet Disorder Support Association
133 Rollins Avenue, #5
Rockville, MD 20852

Phone: 1- 87-PLATELET 877-528-3538 (toll free) or 301-770-6636

Fax: 301-770-6638 - e-mail: pdsa@pdsa.org

Barth Syndrome Foundation 2010 Request for Research Proposals

The Barth Syndrome Foundation, Inc. (BSF) and its international affiliates are pleased to announce the availability of funding for basic science and clinical research on the natural history, biochemical basis, and treatment of Barth syndrome. There are no geographical limitations of this funding.

Background
Barth syndrome (BTHS) is a serious X-linked genetic condition associated with cardiomyopathy, neutropenia, skeletal muscle weakness, exercise intolerance, growth delay, and diverse biochemical abnormalities (including defects in mitochondrial metabolism and phospholipid biosynthesis). Because many clinical and biochemical abnormalities of Barth syndrome remain poorly understood, we are seeking proposals for both basic science and clinical research that may shed light on any aspect of the syndrome. We are determined to find improved treatments—and ultimately a cure—for this rare and under-diagnosed disorder.

Types of Proposals Sought
We are interested in providing “seed grant funding” to young investigators as well as attracting experienced investigators new to the field of BTHS basic science or clinical research. We anticipate that these funds will be used for the testing of initial hypotheses and the collection of preliminary data leading to successful long-term funding by the National Institutes of Health (NIH) and other major granting institutions around the world.

Funding
We anticipate awarding several one– or two–year grants of up to $40,000 each. Funds will be available soon after the successful grant applicants have been notified in early March, 2011.

Process
We have a competitive grant process. Applications should be of 10–15 pages in length and must follow the instructions listed on the BSF website. In general terms, detailed information about the specific aims, significance, research design and methods, personnel, facilities, and budget will be required, along with evidence of application to the relevant Institutional Review Board for any work involving human subjects or to the Animal Use and Protection Committee for any work involving animal subjects.

Completed proposals will be forwarded to the BSF Scientific and Medical Advisory Board (as well as to expert outside reviewers) for evaluation. Based on the recommendations of the BSF Scientific and Medical Advisory Board, the BSF Board of Directors will make the final funding decisions for the grant applications. Once the final funding decisions have been made, BSF affiliates will decide which, if any, of the approved grants they would like to fund.

Deadline
The deadline for submission of completed research grant applications from interested researchers is October 31, 2010. Grants will be awarded in early March, 2011.

Contact Information:
Matthew J. Toth, Ph.D.
Science Director
Barth Syndrome Foundation, Inc.
mtoth[at]barthsyndrome.org
mtothbsf@comcast.net

Conference Student Scholarships: Environmental Epigenomics and Disease Susceptibility

Scholarship Deadline: November 30, 2010 (Midnight US Mountain Standard Time)

There are now compelling human epidemiological and animal experimental data that indicate the risk of developing adult-onset complex diseases and neurological disorders are influenced by persistent epigenetic adaptations in response to prenatal and early postnatal exposures to environmental factors. Epigenetics refers to heritable changes in gene function that occur without a change in the sequence of the DNA. The main components of the epigenetic code are DNA methylation, histone modifications, and non-coding RNAs. The epigenetic programs in cells are normally faithfully reproduced during mitosis. Moreover, they can also be maintained during meiosis, resulting in epigenetic transgenerational disease inheritance, and potentially introducing phenotypic variation that is selected for in the evolution of new species. The objective of this conference is to provide evidence that environmental exposures during early development can alter the risk of developing medical conditions, such as asthma, autism, cancer, cardiovascular disease, diabetes, obesity, and schizophrenia later in life by modifying the epigenome. Epigenetic research promises to markedly improve our ability to diagnosis, prevent, and treat the pathological conditions of humans; however, it also introduces unique legal and ethical issues. These will also be discussed.

Keystone Symposia is offering scholarships to students and post-docs this conference season. These scholarships, of up to $1000 each, are to be used to help defray the expenses associated with conference attendance, including air (on a U.S. air carrier), ground transportation and lodging costs. Receipts will be required to receive reimbursement.

Abstracts submitted for poster presentation will be used as the basis for awarding the scholarships. Conference organizers will select the scholarship recipients based on the quality of science of the abstract and the relevance of the abstract to the conference topic. Only one application per abstract is accepted.

Keystone Symposia
221 Summit Place #272
PO Box 1630
Silverthorne, CO 80498
www.keystonesymposia.org

Financial Assistance / Student Scholarships
Phone: +1 (800) 253-0685 or
Ksenia Shambarger - +1 (970) 262-1230 extension 140
Fax: +1 (970) 262-0311

Progeria Research Foundation Research Grants

The Progeria Research Foundation awards grants to applicants who seek to conduct research to find the cause, treatment, or cure for Hutchinson-Gilford Progeria Syndrome (HGPS).

The next grant application is due October 20th, 2010, for consideration at the Board of Directors' December 2010 meeting.

-- Principal investigators must hold post-doctoral positions or beyond.
-- Awards will be granted only to applicants affiliated with institutions with 501(c)(3) status, or the equivalent for foreign institutions.
-- Proposed projects must have specific relevance to HGPS, and show promise for contributing to the scientific advancement in this field of study.
--Projects will ordinarily be funded for a period of one to two years. Under exceptional circumstances, funding will be continued for a third or fourth year of the project.
--Grant awards will be provided in amounts up to $50,000 per year.

Progeria Research Foundation
P.O. Box 3453
Peabody, MA 01961-3453

For Fed Ex or other deliveries ONLY:
2 Bourbon Street
Suite 208
Peabody, MA 01960

Phone Number: (978) 535-2594

Fax: (978) 535-5849

E-mail info@progeriaresearch.org

Cystic Fibrosis Foundation Clinical Fellowships--1st & 2nd Yr Fellowships

Application Deadline for 1st & 2nd Yr Fellowships: First Wednesday of October
Application Deadline for 3rd, 4th & 5th Yr Fellowships: First Wednesday of September

The intent of this award is to encourage specialized training early in a physician’s career and to prepare candidates for careers in academic medicine. Training must take place in a CF Foundation-accredited care center and must encompass diagnostic and therapeutic procedures, comprehensive care and CF-related research. Applicants must be eligible for Board certification in pediatrics or internal medicine by the time the fellowship begins.

Awards are $47,600 (first year) and $49,250 (second year) for stipend. Also, fellowships are available for additional basic and/or clinical research training in the third, fourth, and fifth years to qualified applicants. Recipients are expected to be subspecialty Board eligible at the completion of the program.

Applicants and sponsors must submit a proposal of the research studies to be undertaken and other specialized training that will be offered during this third year. Up to $68,250 may be awarded: $58,250 for stipend and $10,000 for research costs (indirect costs not allowed).

Recipients who do not enter a career of academic medicine will be subject to payback provisions. U.S. citizenship or permanent resident status is required.

Please direct inquiries to:

Grants and Contracts Office
Cystic Fibrosis Foundation
6931 Arlington Road
Bethesda, MD 20814
(800) FIGHT CF
(301) 951-4422
grants@cff.org

Cure Congenital Muscular Dystrophy Research Grants

Cure CMD Research Grant Goals

Cure CMD research goals reflect our mission to bring research, treatments and in the future, a cure for the congenital muscular dystrophies.

1. Identify new therapeutic targets in the CMDs
2. Identify small molecule compounds that work at these therapeutic target sites with priority towards drugs with existing pediatric FDA approval
3. Facilitate the drug development process to ensure that each positive drug identification with promising preliminary data can be brought rapidly to preclinical trials and subsequent treatment trials
4. Support and endorse clinical research, infrastructure and biomarker discovery required to launch effective and efficient clinical trials in rare disease
5. Advance drug discovery with a diverse treatment pipeline
6. Support new scientific investigators with innovative ideas and a commitment to CMD research

Struggle Against Muscular Dystrophy (S.A.M) CMD Translational Research Grant: Annual Deadline, Sept 15th.

S.A.M is a unique CMD advocacy group launched by two parents whose son has CMD. SAM’s focus is to support and foster CMD research through successful fundraising initiatives. The SAM Translational Research Award delivers upon its promise to target research focused on CMD treatment. The grant is made possible through Cure CMD and SAM collaboration and the Cure CMD research grant funding mechanism.

Cure CMD and SAM invite proposals to award a 1-2 year grant focusing on pre-clinical and clinical investigations that will advance treatment for congenital muscular dystrophy (CMD). The total award is limited to $100,000 (direct costs only). Annual RFA deadline Sept 15th. Grant application above. On the First Page of the Cure CMD grant application, please specify S.A.M. CMD Translational Research Grant

Cure CMD General Research Grants: Annual Deadline Sept 15th.

Elisabeth Giauque Short Term CMD Research Grant: Cure CMD provides a short-range or “seed” grant (usually $25,000- $50,000 per year for 1-2 years, direct costs only) to attract new investigators to the CMD field and assist existing investigators to support early phases of their research. The short-range grant focus is to collect preliminary data and test initial hypotheses. The short-range grant goal is to translate successful preliminary research to either second tier Cure CMD midrange grants (R-21 type) or third tier, long range (R-01 grants, 2-5 years) from outside funding sources, such as the National Institutes of Health. Annual RFA deadline, Sept 15th. Grant application above. On the first page of the Cure CMD grant application, please specify Elisabeth Giauque Short Term CMD Research Grant.

info@curecmd.com

Cure CMD
P.O. Box 701
Olathe, KS 66051
Ph. 1-866-400-3626