Grants
IQSEC2 Research and Advocacy Foundation Research Grant Program 2023 full application (by invitation only)
The IQSEC2 Research and Advocacy Foundation will provide a 1-year grant, with a no cost extension of up to one year granted with approval, to support research related to IQSEC2-related disorder. The foundation will award either one grant of $60,000 or two grants for $30,000.
Background
IQSEC2 is a genetic condition that causes intellectual disability and sometimes other physical, neurological, or psychiatric symptoms. People with this condition can have seizures that are often difficult to control with medications. Other symptoms may include motor and language development delay, regression of learning abilities, autistic-like behavior, characteristic hand movements, and behavioral problems. Physical features may include abnormal head shape (plagiocephaly), very small head (microcephaly), reduced muscle tone (hypotonia), and crossed eyes (strabismus). This condition is caused by mutations in the IQSEC2 gene, which is located on the X-chromosome. Depending on the severity of the gene mutation, the features can range from only intellectual disability to a syndrome that includes the other symptoms. In general, males are more affected than females. Most cases are not inherited from a parent but are caused by a new (de novo) mutation. There is no specific treatment, but early intervention and other services can support development.
The IQSEC2 Research and Advocacy Foundation, in collaboration with the Orphan Disease Center, is seeking grant applications that will address both symptoms of the disease (i.e. intractable seizures) and/or mechanistic studies that are oriented towards developing therapies addressing the underlying cause of IQSEC2 mediated intellectual disability, autism and epilepsy. The grant will exclusively be on IQSEC2 mutation mediated disease and not on other disease mutations that may have some overlap. The grant is intended to serve as a seed for additional funding from other granting agencies and part of the application will need to contain a section where the researchers discuss how they will do this. In addition, it will be critical for the researcher to suggest how this grant will fit into the strategic goals of the IQSEC2 foundation towards reducing morbidity and developing novel treatments of IQSEC2 mediated disease. Innovative therapies are particularly welcome.
Eligibility
All individuals holding a faculty-level appointment at an academic institution or a senior position at a non-profit institution or foundation are eligible to respond to this RFA. Researchers with no prior experience with IQSEC2 are encouraged to apply.
Full Applications are Due Friday, December 22, 2023 by 8pm EST
To Apply:
Step 1: Please review the RFA Guidelines.
Step 2: Download and complete the below forms:
Step 3: Apply through Submittable
For any scientific inquiries regarding this grant please email Deborah Requesens.
For any administrative inquiries regarding this grant please email Leslie Silverman.
IQSEC2 Research and Advocacy Foundation Research Grant Program 2023
The IQSEC2 Research and Advocacy Foundation will provide a 1-year grant, with a no cost extension of up to one year granted with approval, to support research related to IQSEC2-related disorder. The foundation will award either one grant of $60,000 or two grants for $30,000.
Background
IQSEC2 is a genetic condition that causes intellectual disability and sometimes other physical, neurological, or psychiatric symptoms. People with this condition can have seizures that are often difficult to control with medications. Other symptoms may include motor and language development delay, regression of learning abilities, autistic-like behavior, characteristic hand movements, and behavioral problems. Physical features may include abnormal head shape (plagiocephaly), very small head (microcephaly), reduced muscle tone (hypotonia), and crossed eyes (strabismus). This condition is caused by mutations in the IQSEC2 gene, which is located on the X-chromosome. Depending on the severity of the gene mutation, the features can range from only intellectual disability to a syndrome that includes the other symptoms. In general, males are more affected than females. Most cases are not inherited from a parent but are caused by a new (de novo) mutation. There is no specific treatment, but early intervention and other services can support development.
The IQSEC2 Research and Advocacy Foundation, in collaboration with the Orphan Disease Center, is seeking grant applications that will address both symptoms of the disease (i.e. intractable seizures) and/or mechanistic studies that are oriented towards developing therapies addressing the underlying cause of IQSEC2 mediated intellectual disability, autism and epilepsy. The grant will exclusively be on IQSEC2 mutation mediated disease and not on other disease mutations that may have some overlap. The grant is intended to serve as a seed for additional funding from other granting agencies and part of the application will need to contain a section where the researchers discuss how they will do this. In addition, it will be critical for the researcher to suggest how this grant will fit into the strategic goals of the IQSEC2 foundation towards reducing morbidity and developing novel treatments of IQSEC2 mediated disease. Innovative therapies are particularly welcome.
Eligibility
All individuals holding a faculty-level appointment at an academic institution or a senior position at a non-profit institution or foundation are eligible to respond to this RFA. Researchers with no prior experience with IQSEC2 are encouraged to apply.
Letter of Intereset (LOI) Applications are Due Friday, November 3, 2023 by 8pm EST
To Apply:
Step 1: Please review the RFA Guidelines.
Step 2: Apply through Submittable
For any scientific inquiries regarding this grant please email Deborah Requesens.
For any administrative inquiries regarding this grant please email Leslie Silverman.
Million Dollar Bike Ride 2023 Pilot Grant Program - Full Application (by invitation only)
The ODC is offering 39 research opportunities focusing on 31 different rare diseases. This program provides a one-year grant to support research related to a rare disease represented in the 2023 Million Dollar Bike Ride. Number of awards and dollar amounts vary per disease based on fundraising totals by each disease team.
The MDBR 2023 Pilot Grants are open to the international community holding a faculty‐level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation
Full Applications (by invitation only) are Due Monday, October 16, 2023 by 8PM EST.
To Apply:
Step 1 - Please review the 2023 MDBR Pilot Grant Program RFA Guidelines.
Step 2 - Download and complete the below forms:
Step 3 - Complete the Application Form on Submittable.
Research Areas for the 2023 MDBR Grant Program:
Million Dollar Bike Ride 2023 Pilot Grant Program - LOI (Copy)
The ODC is offering 39 research opportunities focusing on 31 different rare diseases. This program provides a one-year grant to support research related to a rare disease represented in the 2023 Million Dollar Bike Ride. Number of awards and dollar amounts vary per disease based on fundraising totals by each disease team.
Letter of Interest (LOI) Applications are Due Friday, September 15th, 2023 by 8pm ET
To Apply:
Please review the Request for Applications (RFA)
Complete the Application Form on Submittable.
The MDBR 2023 Pilot Grants are open to the international community holding a faculty‐level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation.
Prior MDBR award recipients must have current and updated project reporting to be eligible for selection.
Research Areas for the 2023 MDBR Grant Program:
Million Dollar Bike Ride 2023 Pilot Grant Program - LOI
The ODC is offering 39 research opportunities focusing on 31 different rare diseases. This program provides a one-year grant to support research related to a rare disease represented in the 2023 Million Dollar Bike Ride. Number of awards and dollar amounts vary per disease based on fundraising totals by each disease team.
LOI Extension for select disease (see below for list of diseases)
Letter of Interest (LOI) Applications are extended for select disease & Due Friday, October 6th, 2023 by 8pm ET
To Apply:
Please review the Request for Applications (RFA)
Complete the Application Form on Submittable.
The MDBR 2023 Pilot Grants are open to the international community holding a faculty‐level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation.
Prior MDBR award recipients must have current and updated project reporting to be eligible for selection.
EXTENDED LOI DUE DATE IS FOR THE BELOW DISEASE TEAMS
Research Areas for the 2023 MDBR Grant Program:
Bow Foundation 2023 Grant Program Full Application (by invitation only)
The Bow Foundation, will provide a 1-year grant to support research related to GNAO1 Neurodevelopmental Disorder. Up to 2 awards will be granted at $100,000 each.
Background
GNAO1 (G Protein Subunit Alpha O1) is a protein coding gene. Most patients with a GNAO1 neurodevelopmental disorder are diagnosed as infants or young children. Many of the patients begin experiencing seizures, abnormal movements and developmental delays in their infancy. The seizures are often refractory, and the patients seem to cycle through numerous medications as they try to find relief. The abnormal movements seem to worsen as the patients age. Several patients have benefited from deep brain stimulation surgery, including the youngest patient in the world to receive the procedure (a 2-year-old in the UK).
The Orphan Disease Center, in collaboration with the Bow Foundation, is seeking grant applications that aim to further progress our understanding of the disease, the available therapeutic options, and investigating strategies to establish outcome measurements. The RFA could focus on one, or several, of the following aims to further advance GNAO1 research and therapeutic approaches:
Novel therapeutic approaches, including, but not limited to, techniques in genome editing, RNA-based mechanisms, biologics, novel cell-based therapeutics, and development of novel therapeutic compounds, including through small molecule repurposing or screening against validated phenotypes in human cellular systems.
Identification of short-term biomarkers that can monitor disease activity and treatment response.
Establishment of outcome measures for future clinical trials.
Development of therapeutic approaches in early symptomatic patients.
Supporting pilot clinical trials, preclinical trials, or animal model trials that promote drug repurposing strategies.
Development of a standardized evaluation criteria for clinical projects allowing uniformity of patients as well as the severity and progression of the disease.
Development of cellular models (i.e. oligodendrocytes) for evaluation of therapeutic options to translate for clinical use.
Eligibility
All individuals holding a faculty-level appointment at an academic institution or a senior position
at a non-profit institution or foundation are eligible to respond to this RFA.
Full Applications are Due Friday, April 14, 2023 by 8pm EST
To Apply:
Step 1: Please review the RFA Guidelines.
Step 2: Download and complete the below forms:
Step 3: Apply through Submittable
For any scientific inquiries regarding this grant please email Deborah Requesens.
For any administrative inquiries regarding this grant please email Leslie Silverman.
2023 CDKL5 Pilot Grant Program Full Application (by invitation only)
The ODC and Loulou Foundation CDKL5 Pilot Grant Program provides a one‐year grant for $150,000.00 (total cost) to support research related to CDKL5 Deficiency Disorder (CDD). The number of awards is not fixed, and may vary.
Full Applications (by invitation only) are Due Friday, March 24th, 2023 by 8 PM EST.
To Apply:
Step 1 - Please review the "Full Application Instructions and Review Procedure" section of the 2023 CDKL5 Program of Excellence Pilot Grant Program RFA Guidelines.
Step 2 - Download and complete the forms below:
Step 3 - Complete the Application Form on Submittable.
To review the Loulou Foundation's patent policy please click here
For any scientific inquiries regarding this grant please email Dan Lavery.
For any administrative inquiries regarding this grant please email Samantha Charleston.
ADLD Center Grant Program Full Application (by invitation only)
ADLD Center Grant Program
The ADLD Center, in collaboration with the Orphan Disease Center, will provide a 1-year grant to support research related to Autosomal Dominant Leukodystrophy (ADLD). Up to 2 awards will be granted at $50,000 each.
Background
Adult-onset Autosomal Dominant Leukodystrophy (ADLD) is a rare genetic disorder that develops symptoms in the fourth or fifth decade due to nerve damage that slowly progresses. The onset often includes autonomic conditions such as bowel or bladder dysfunction, male impotence, loss of fine motor skills, and orthostatic hypotension. Later symptoms include difficulties using and controlling legs and arms, ultimately leading to paralysis and problems swallowing, and eventually developing into intellectual impairment. ADLD is a fatal disorder, but it is slowly progressive; patients often survive for several decades.
ADLD can be caused by one of two genetic defects - either a duplication of the gene LMNB1 that encodes for the protein Lamin B1, or an upstream deletion. Lamin B1 is a protein that is present in every cell in the body and plays an essential role in cell structure. Either of the two aforementioned genetic changes results in toxic overexpression of Lamin B1. Although Lamin B1 is present in all cells in the body, overexpression of Lamin B1 mainly causes problems in the central nervous system where it causes demyelination.
The Orphan Disease Center, in collaboration with the ADLD Center, is seeking grant applications for multidisciplinary teams of scientists that aim to further progress our understanding of the disease, the available therapeutic options, and investigating strategies to establish outcome measurements. The RFA could focus on one, or several, of the following aims to further advance ADLD research and therapeutic approaches:
Identification of short-term biomarkers that can monitor disease activity and treatment response.
Establishment of outcome measures for future clinical trials.
Development of therapeutic approaches in early symptomatic patients.
Supporting pilot clinical trials, preclinical trials, or animal model trials that promote drug repurposing strategies.
Development of a standardized evaluation criteria for clinical projects allowing uniformity of patients as well as the severity and progression of the disease.
Development of cellular models (i.e. oligodendrocytes) for evaluation of therapeutic options to translate for clinical use.
Evaluate pre-clinical patients (MRIs, genetic testing).
Eligibility
All individuals holding a faculty-level appointment at an academic institution or a senior position at a non-profit institution or foundation are eligible to respond to this RFA.
Full Proposal by invitation only
Please submit your full application documents by February 24, 2023.
To Apply:
Step 1 - Review RFA
Step 2 - Download and complete the below forms:
Step 3 - Apply through submittable
For any scientific inquiries regarding this grant please email Debbie Requesens
For any administrative inquiries regarding this grant please email Samantha Charleston.
2023 CDKL5 Pilot Grant Program LOI
The ODC and Loulou Foundation CDKL5 Pilot Grant Program provides a one‐year grant for $150,000.00 (total cost) to support research related to CDKL5 Deficiency Disorder (CDD). The number of awards is not fixed, and may vary.
Letter of Intent (LOI) are Due Friday, February 24, 2023 by 5pm EST
To Apply:
Please review the "LOI Instructions and Review Procedure" section of the 2023 CDKL5 Program of Excellence Pilot Grant Program RFA Guidelines.
2. Complete the Letter of Intent (LOI) Application Form on Submittable.