Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

Inclusion Criteria:

1. Participants with a clinical diagnosis of Type 3 SMA.

2. Participants who are ambulatory, defined as being able to walk at least 50 metreswithout walking aids at screening during the 6-minute walk test.

3. Participant with genetic confirmation of diagnosis (e.g., homozygous deletion orcompound heterozygous deletion and mutation of survival of motor neuron 1 gene [SMN1])

4. Participant with 3 to 5 copies of survival of motor neuron 2 gene [SMN2].

5. Participant has a body mass index (BMI) within the range 19-35 kg/m2 (inclusive).

6. Participant is male or female.

7. Contraceptive use by men and women must be consistent with local regulationsregarding the methods of contraception for those participating in clinical studies.

8. Participant is capable of giving signed informed consent which includes compliancewith the requirements and restrictions listed in the informed consent form (ICF) andin the protocol.

Exclusion Criteria:

1. Participants with prior surgery or fixed deformity (scoliosis, contractures) whichwould restrict ability to perform study-related tasks.

2. Participants with other significant disease that may interfere with theinterpretation of study data (e.g., other neuromuscular or muscular diseases).

3. Participants with other significant clinical and/or laboratory safety findings thatmay interfere with the conduction or interpretation of the study

4. Participants received treatment with an investigational medical product (IMP) within 30 days (or 5 half-lives of the medication, whichever is longer) prior to Day 1.

5. Participants with history of poor compliance with relevant SMA therapy.