In an agreement meant to provide Ionis Pharmaceuticals with additional money to advance potential therapies for various diseases, the company is giving Royalty Pharma a percentage of the payments it receives from sales of Spinraza (nusinersen), an approved therapy for spinal muscular atrophy (SMA). In exchange, …
The ongoing SAPPHIRE clinical trial testing apitegromab, Scholar Rock’s investigational muscle-directed therapy, in people with spinal muscular atrophy (SMA) is expected to complete patient enrollment later this year, the company announced. Top-line data from the trial are anticipated in 2024. Should results be positive, Scholar Rock expects…
CANbridge Pharmaceuticals has acquired exclusive global rights to develop, manufacture, and commercialize a second-generation gene therapy for spinal muscular atrophy (SMA). The company had completed preclinical studies of the investigational gene therapy in collaboration with the Horae Gene Therapy Center at the University of Massachusetts (UMass) Chan…
Biogen is collaborating with Alcyone Therapeutics to develop an implantable medical device that more easily delivers antisense oligonucleotide (ASO) therapies to the spinal canals of people with certain neurological disorders. Under the terms of the agreement, Biogen will acquire exclusive global rights to Alcyone’s ThecaFlex DRx system in…
The U.S. Food and Drug Administration (FDA) has given NMD Pharma the green light to conduct a Phase 2 trial of its oral therapy NMD670 in people with spinal muscular atrophy (SMA) type 3. The upcoming clinical trial aims to evaluate the efficacy, safety, and tolerability of…
Researchers have developed and validated four new cell lines derived from four patients with spinal muscular atrophy (SMA) with lower extremity predominance (SMA-LED) that can be used as a tool in studies of this rare form of spinal muscular atrophy. With the characteristics of the patient it was…
Among adults with spinal muscular atrophy (SMA), men show signs of greater disease severity than women, particularly as young adults, according to a recent study. For patients with SMA type 3, a greater proportion of women were able to maintain their ability to walk compared with men. “Gender should…
The incidence of infantile spinal muscular atrophy (SMA) has increased in Japan since 2016 and this could be related to greater disease awareness among clinicians, earlier diagnoses, and the introduction of new treatments — although this relationship remains to be established, a study has found. On the large Japanese…
For a boy with spinal muscular atrophy (SMA) type 2, wearing a robotic gait-training device at home led to mobility gains and enabled him to participate in new activities like games and sports, a case study has found. According to a nursing assessment, the training was well-tolerated, with only…
Levels of the hunger hormone leptin are not associated with motor function in children and adolescents with spinal muscular atrophy (SMA) types 2 and 3, according to a Serbian study. Higher leptin was linked to a greater body mass index (BMI) — a surrogate marker of body fat —…
