The Speak Foundation
The Speak Foundation
Are you newly diagnosed with limb-girdle muscular dystrophy? If so, your starting point is here. This webinar explains the basics of LGMD.
The C.A.R.E. Program's acronym stands for "Connecting and Reaching Everyone" with limb-girdle muscular dystrophy. This program offers educational webinars with information from the pharmaceutical and biotech industry directly to patients. Additionally, this program partners with top level researchers in some of the world's best universities from GRASP LGMD to present the best information on LGMD. Cutting-edge research and information about clinical trials with new treatments are presented in our webinars to help individuals with LGMD feel connected and informed.
A hallmark feature of this program is our diligence to locate newly diagnosed patients living with limb-girdle muscular dystrophy. To do this, we have developed the LGMD News magazine which has in every edition, a section for newly diagnosed individuals that directs them on where to go to find additional resources about their unique subtype. The magazine is distributed worldwide, sent directly to U.S. patients' homes and clinics where LGMD individuals are diagnosed, and sent via digital download to our international community. This bridges the gap for doctors and allows them to share an important resource guide with patients and families struggling to find answers.
Healthcare providers often talk about “outcome measures” during clinic visits or as part of research trials. What is their purpose and why do they matter? This webinar reviews common outcome measures, why they’re used, and why they can be so important for patients, clinicians, and researchers. The webinar is geared towards those living with LGMD and helps them to understand the basics of outcome measures.
Dr. Lindsay Alfano is a physical therapist with expertise in the care and evaluation of patients with neuromuscular disease including outcome measure development, evaluation, and administration. She plays an integral role in planning and designing clinical trials and contributes to protocol development, outcome measure selection, statistical analysis, and interpretation for ongoing clinical trials. Dr. Alfano serves on an international consortium of neuromuscular physical therapy experts that provide industry-standard training and reliability for multisite international trials. Her research goals focus on standardizing training in rare disease, as well as developing novel outcome tools to measure change in movement abilities while minimizing the burden of testing.
Many individuals with LGMD have genetic test reports that are complicated. Despite knowing the specific genes that cause LGMD subtypes, diagnostic uncertainty remains for many patients. We know that over 50% of patients with LGMD receive unclear results from their genetic testing. In this educational webinar, we share important information about genetic test results. This is Part 1 of a two-part, educational webinar on this topic.
This is Part 2 of our series on genetic test results, "Genetic Test Reports and Understanding VOUS", with Monkol Lek, PhD. Understanding VOUS is something that individuals with LGMD symptoms find confusing. This educational webinar helps you to understand more about genetic test results.
This is the first in a series of educational webinars with The Speak Foundation in conjunction with GRASP LGMD and other partners. Dr. Nick Johnson is our honored guest speaker and he is sharing on the the effects of COVID-19 on clinical trials for limb-girdle muscular dystrophy.
ML Bio Solutions is advancing the first-ever oral treatment for LGMD2i – BBP-418 (Ribitol). This treatment may enhance, and partially compensate for, diminished muscle function caused by the genetic driver of the disease. Our panelists are Douglas Sproule, MD (Chief Medical Officer, ML Bio Solutions) and Nicholas Johnson, MD (Principle Investigator for the BBP-418 Lead-in Study).
This webinar highlights potential gene repair approaches with LGMD 2i and 2g. This webinar was held on September 18th, 2020, with CureLGMD2i. Dr. Charles Emerson and Dr. Scot Wolfe from UMass explained gene editing in this informative presentation. They are doing amazing research out of University of Massachusetts for LGMD 2i and 2g. THANK YOU to CureLGMD2i for sponsoring this exciting webinar!
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The Limb Girdle Muscular Dystrophy Foundation
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The Speak Foundation is a 501(c)(3), Est. 2008.
Sign up to be included in the Limb Girdle Muscular Dystrophy Patient Network where you will receive cutting edge research updates from the leading experts in the world such as the GRASP LGMD consortium and also be invited to hear about new clinical trials. When you sign up here, you will also receive the free LGMD News Magazine if you live in the USA. Anyone can join the LGMD Patient Network.