At the 2023 MDA’s Clinical and Scientific conference, the neurology resident at Penn Medicine talked about the challenges of delivering intrathecal therapies and the importance of planning clinical resources for patients with ALS. [WATCH TIME: 3 minutes]
WATCH TIME: 3 minutes
“As we anticipated, for the first potential approval of [tofersen], we wanted to make sure that we had the right clinical resources in place to prevent delays to care. We realize that with intrathecal therapies, as well as genetic therapies, there are often additional clinical resources that cause a need to have pre-established collaborations prior to drug approval.”
In genetically predisposed neuromuscular conditions, potential therapies may require additional clinical resources that are necessary for providers to provide timely care to their patients. Thus, with potential genetic therapies on the horizon, it may be important to consider the best method of care delivery for these treatments and the most effective ways for patients to receive quality care in clinical practice.
At the recent 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas, Jennifer Morganroth, MD, MBA, presented a talk during a session centered on gene therapies in amyotrophic lateral sclerosis (ALS).1 In her presentation, she focused specifically on the measures taken with Biogen’s tofersen, an investigational antisense oligonucleotide currently under review for SOD1 mutated-mediated ALS. As of now, there are no FDA approved therapies to treat familial forms of ALS, including SOD1-ALS, which may change with tofersen’s PDUFA date approaching soon on April 25, 2023.2
Morganroth sat down in an interview with CGTLive®, a sister publication to NeurologyLive®, at the conference to provide a brief overview of her presentation, while also elaborating on a few of the other talks given during the session. As new gene therapies are potentially on the horizon, Morganroth, a PGY3 neurology resident at Penn Medicine in the University of Pennsylvania Health System, talked about some of the challenges and unmet needs that she sees in the field. Additionally, she briefly spoke about the potential implications for patients if tofersen is approved.
Editor’s Note:Morganroth disclosed that she participated in an MD Co-op for the Neuromuscular Clinical Development Unit at Biogen as a paid 5 week elective over the summer of 2022.