Current and emerging therapies and treatment approaches can offer symptom relief and improved quality of life for patients with metachromatic leukodystrophy (MLD). But given MLD’s rarity and health care professionals’ lack of familiarity with the disease and its treatment, diagnosis is often inaccurate or delayed, negatively impacting outcomes.
During this 90-minute enduring material, expert panelists will discuss biomarkers that identify patients at the pre-symptomatic phase of the disease and share knowledge about caring for patients with MLD.
At the end of this CME/CE activity, participants should be able to:
Supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.
Physicians, physician associates (PAs), nurse practitioners (NPs), nurses and pharmacists specializing in genetics, metabolism, neurology, pediatrics, internal medicine and/or primary care
It is the policy of CME Outfitters, LLC, to ensure independence, balance, objectivity, and scientific rigor and integrity in all of their CE activities. Faculty must disclose to the participants any relationships with commercial companies whose products or devices may be mentioned in faculty presentations, or with the commercial supporter of this CE activity. CME Outfitters, LLC, has evaluated, identified, and mitigated any potential conflicts of interest through a rigorous content validation procedure, use of evidence-based data/research, and a multidisciplinary peer review process.
Dr. Eichler reports the following financial relationships: Advisory Board: Bluebird Bio, Inc. and SwanBio Therapeutics, Inc. Consultant: Autobahn Therapeutics; Poxel S.A.; SwanBio Therapeutics, Inc.; Takeda Pharmaceuticals U.S.A., Inc.; Taysha Gene Therapies; and UpToDate, Inc. Grants: European Leukodystrophy Association; National Institute of Neurological Disorders and Stroke (NINDS); and Undiagnosed Disease Network (UDN). Research Support: Aspa Therapeutics; Bluebird Bio, Inc.; and Sio Gene Therapies. Speakers Bureau: American Society for Gene and Cell Therapy (ASGCT) and PRIME Education, LLC. Stock Shareholder (ownership interest): SwanBio Therapeuitics, Inc. Other Financial or Material Support: UpToDate, Inc. Authorship Fees
Dr. Adang reports the following financial relationships: Consultant: MEGMA; Orchard Therapeutics; and Takeda Pharmaceuticals U.S.A., Inc. Grants: MSD Action Foundation; National Center for Advancing Translational Sciences (NCATS); National Institute of Neurological Disorders and Stroke (NINDS); and The Family Foundation.
Ms. Hickey reports no financial relationships to disclose.
The following peer reviewer and CME Outfitters staff have no financial relationships:
Faculty of this CE activity may include discussions of products or devices that are not currently labeled for use by the FDA. The faculty have been informed of their responsibility to disclose to the audience if they will be discussing off-label or investigational uses (any uses not approved by the FDA) of products or devices.