National Tuberous Sclerosis Association Inc

“The form 990 is reviewed, in detail, by the board of directors' audit committee. Recommendations are made by the committee members for any changes/edits/additions. After those have been incorporated, the audit committee votes whether to approve and then forward the 990 to the finance and executive committees. The finance and executive committees perform the final review and then vote whether to approve on behalf of the board of directors. A copy of the approved 990 is shared with the entire board prior to its filing with the irs.”

“Membership is available to any person who subscribes to the purposes and objectives of the corporation, without regard to race, religion, gender, sexual orientation, age, color, national origin or mental or physical handicap or disability. There shall be no limit to the number of members in the corporation. 1) there may be one or more classes of membership as determined by the board. 2) membership is not transferable or assignable.”

“The tsc alliance is a membership-based organization, which means members help elect the board of directors. The tsc alliance membership program allows individuals to state their intent to be a member for the purpose of governance. There were no levels to membership in 2021. Anyone can be a member at no cost.”

“The form 990 is reviewed, in detail, by the board of directors' audit committee. Recommendations are made by the committee members for any changes/edits/additions. After those have been incorporated, the audit committee votes whether to approve and then forward the 990 to the finance and executive committees. The finance and executive committees perform the final review and then vote whether to approve on behalf of the board of directors. A copy of the approved 990 is shared with the entire board prior to its filing with the irs.”

“Annually each member of the board of directors will receive notice of the organization's conflict of interest statement. Each member will be provided with a statement to make disclosure of any potential conflict of interest. If during the course of the year a potential conflict of interest arises that has not previously been disclosed, the board member will make written notice of a potential conflict of interest and recuse himself or herself from any discussions and votes in connection with the issue identified. Any time a member is recused from discussion on an issue, the minutes of committee meeting and board meeting will duly record such actions. The following potential conflicts of interest were disclosed for 2021: board member mustafa sahin, ph.d., m.d., is employed at boston children's hospital, which received $113,659 in grants and $9,015 in fees for services for participation in the tsc natural history database. Board member darcy krueger, md, mpa, is employed at the cincinnati children's hospital medical center, which received a $100,000 grant payment for his work on stop-2 and $8,190 in fees for services for participation in the tsc natural history database. Board member jessica krefting, rn, is employed at university of alabama at birmingham, which received $7,715 in fees for services for participation in the tsc natural history database. Board member and chair peter crino, md, phd, is chair of the department of neurology at the university of md, which recieved a $18,634 grant payment.”

“The compensation committee reviews and approves the salaries of the president/ceo, chief scientific officer, cfo, and any employee appearing on the form 990, in accordance with the tsc alliance bylaws. Such review and approval occurs initially upon hiring, upon annual reviews, and whenever modified. The organization's executive remuneration has been structured to ensure that it: is reasonable; provides a competitive compensation program to retain, attract and reward key employees and achieves clear alignment between total remuneration and delivered business and personal performance over the short and long-terms. The compensation is reviewed by the compensation committee to ensure: - comparability, - proper review, and - substantiation in setting the compensation.”

“All governing documents, conflict of interest policy and financial statements are available upon written request.”

“The preclinical consortium invites compound nominations from academic and industry researchers to continue to refresh our pipeline based on the latest data and novel ideas. The nominations are peer-reviewed by consortium members and prioritized by the preclinical consortium steering committee for funding by the tsc alliance. If the tsc alliance funds the study, the data are shared with the consortium for transparency and stimulation of additional hypotheses. An annual face-to-face members meeting is typically held each year in april to keep members engaged and aligned on the consortium's strategy and priorities. Partnership with pharmaceutical companies is an important aspect of the consortium, as these organizations have the infrastructure to efficiently move preclinical research findings to clinical testing and eventually commercial distribution. As of december 2021, 10 companies were active members of the consortium. The consortium has had 17 company members since 2016. There is natural turnover of industry membership, principally driven by research data generated by testing their drugs. Due to intellectual property considerations, most companies fully fund their studies through the tsc alliance, which allows the company to keep its data confidential. These confidential studies also incur a 10% overhead paid by the company, which is a source of revenue for the tsc alliance. Since inception, the consortium has helped advance the evaluation of drugs for tsc by conducting 90 studies, evaluating 57 candidate drugs. In 2020, the tsc preclinical consortium evaluated 9 candidate compounds and conducted 22 experiments. In 2012, the tsc alliance helped create the tsc clinical research consortium in partnership with investigators running clinical studies to ensure clinical research in tsc is as efficient and effective as possible. Since then, tsc clinical research consortium investigators have been awarded more than $39 million by the national institutes of health (nih) and food and drug administration (fda) through competitive grant processes. Tsc alliance personnel serve on the leadership team for the consortium, actively track enrollment, and raise community awareness to help identify potential participants for clinical studies. Tsc alliance also provides supplemental financial support to accelerate or expand nih-funded studies. The prevent trial - preventing epilepsy using vigabatrin in infants with tsc - is the first preventative trial in the united states for any form of epilepsy and has completed enrollment of 86 participants. We expect the first results in the first quarter of 2022 and final results in early 2023. This trial builds directly upon the clinical research consortium's first clinical study, which identified specific eeg changes in infants with tsc prior to the onset of seizures. In the prevent trial, infants with tsc receive regular eeg monitoring, and those who develop eeg abnormalities are placed on either vigabatrin or placebo. Any infant who subsequently develops clinical seizures is immediately placed on standard treatment. This trial will determine whether treatment with vigabatrin prior to the onset of clinical seizures in tsc is beneficial to children's developmental and neurologic outcomes. More details about this study are online at www.clinicaltrials.gov/ct2/show/nct02849457. Immediately after the prevent trial enrollment completed, a similar clinical trial began enrolling newborns with tsc before the onset of seizures to test the effects of sirolimus on preventing or delaying seizure onset. The stopping tsc onset and progression 2 (stop-2) trial enrolled five infants at cincinnati children's hospital medical center as an initial safety study. The second stage of the trial is now called "sirolimus tsc epilepsy prevention study (tsc-steps) and the tsc alliance has contributed $200,000 to enable the trial to be expanded to additional sites across the country. The tsc alliance is also providing supplemental”