
E111125: Legacy Lecture Series: Krishna Saxena, MD Endocrine Lecture: Recent therapeutic advances in achondroplasia
This is a one hour recording of the Krishna Saxena, MD Endocrine Lecture presented by Carlos A. Bacino, MD, FACMG.

Dr. Bacino is a Professor, Vice Chair Clinical Affairs, Department of Molecular and Human Genetics, Baylor College of Medicine;
Chief, Genetics Service, Director, Angelman Syndrome Clinic, Texas Children's Hospital;
Medical Director, Baylor Genetics Cytogenetics Laboratory
Dr. Bacino has worked in the field of clinical genetics for over 30 years. His training has provided him with a vast experience in the fields of clinical genetics, cytogenomics and molecular testing. Over the past decade he has been more interested in the detection, delineation and treatment of patients with rare disorders driven by the recent technological. He is the co-PI for the Undiagnosed Disease Network NIH initiative at the Houston clinical site currently in its 11th year. In that capacity he has been involved in the evaluation of patients with rare disorders, many of them successfully diagnosed through genome sequencing and transcriptomics and in many cases aiding to the discovery of new genes. More recently he has become a co-investigator in an NIH sponsored trial known as Texome that provides clinical evaluations and exome sequencing to underserved populations. He has been working in clinical care and research of patients with skeletal dysplasias. He is the PI for multiple multinational clinical research efforts in the achondroplasia field. Dr. Bacino has also worked in the care of Angelman syndrome patients for 20 years and currently the PI for a clinical trial using antisense oligonucleotide therapies in Angelman syndrome through the sponsorship of IONIS Pharmaceutical and more recently the PI for an AAV mediated gene therapy trial in Angelman syndrome.

Krishna Saxena, MD Endocrine Lecture
This lectureship is one that does more than honor a talented physician. It tangibly perpetuates Dr. Saxena’s life-long commitment to high-quality medical education for aspiring pediatricians and pediatric specialists.
This is a forum for the advancement of knowledge, with the hope that it will bring improved health for our community’s children.
Target Audience
Healthcare clinicians caring for pediatric patients and their families.
Learning Objectives
- Illustrate the challenges of achondroplasia co-morbidities and how to follow them clinically.
- Discuss what molecular pathways have taught us for treatment approaches in rare bone disorders.
- Summarize the new advances in achondroplasia treatment therapies as well as upcoming research in the field.
PRESENTER: Carlos A. Bacino, MD, FACMG
FACULTY DISCLOSURE ANNOUNCEMENT
It is our intent that any potential conflict should be identified openly so that the listeners may form their own judgments about the presentation with the full disclosure of the facts. It is not assumed any potential conflicts will have an adverse impact on these presentations. It remains for the audience to determine whether the speaker’s outside interest may reflect a possible bias, either the exposition or the conclusions presented.
Planning committee members and presenter(s) have disclosed they have no relevant financial relationships with ineligible companies whose primary business is producing, marketing selling re-selling, or distributing healthcare products used by or on patients and have disclosed that no conflict of interest exists with the presentation/educational event.

Accreditation Statement:
In support of improving patient care, Children’s Minnesota is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.
Children’s Minnesota takes responsibility for the content, quality and scientific interest of its accredited continuing education events.
Credit Statement:
AMA
Children’s Minnesota designates this enduring activity for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent to their participation in the activity.
ANCC
Children's Minnesota designates this enduring activity for a maximum of 1.0 ANCC nursing contact hour. Nurses should claim only the credit commensurate with the extent of their participation in the activity.
This program offers 1.0 contact hours of continuing education. Most medical professional organizations accept AMA PRA Category 1 Credits™. It is the individual’s responsibility to determine whether an education activity meets the continuing education requirements of their respective licensures and/or professional boards. Go to your respective organization’s website for more detailed information regarding credit requirements.
Available Credit
- 1.00 AMA PRA Category 1 Credit™
- 1.00 ANCC
- 1.00 Attendance
- 1.00 Approved AMA PRA Category 1 Credit™
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Required Hardware/software
You will need an internet connection using either a phone, iPad, or computer.

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