Cure RTD

While Cure RTD remains focused on better treatments and ultimately a cure for RTD, we are also continuously reviewing therapies that could help improve the damage already caused by the disease.

One promising therapy Cure RTD has on our radar is apitegromab, a drug designed to target muscle weakness.

Apitegromab was developed for spinal muscular atrophy, or SMA, where progressive motor weakness is a major feature. It works by blocking myostatin, a natural protein that limits muscle growth and strength. The goal is to improve muscle function, not to treat the underlying genetic cause of SMA (or RTD).

That is why this drug is interesting for RTD.

SMA and RTD are different diseases, but both can leave patients with significant motor weakness, even after treatment. In SMA, weakness comes from loss of motor neuron input to muscle similar the RTD. This overlap gives hope that a drug improving muscle strength in SMA could potentially provide benefit for some RTD patients as well.

The FDA (USA) and EMA (Europe) have accepted the apitegromab application for approval with the drug launch to SMA patients expected later this year.

Apitegromab has not yet been studied or approved for RTD. With the approval and launch, Cure RTD will be able to fund research studies with RTD patients to see whether this approach could help improve strength, mobility, breathing reserve, hand function, or quality of life in RTD patients living with residual weakness.

This is not a cure for RTD, but it may be one more door opening.