Rare disease affects toddler’s mobility
Two-year-old Lincoln Woodmass was diagnosed with SMA type 3 earlier this year after his family had concerns over his walking ability.
There are about 75 known cases in New Zealand and currently no treatment available.
Dr Collette Bromhead, chief executive for NZORD, said in December the US Food and Drug Administration approved a drug called Spinraza, made by Biogen, for the treatment of SMA.
Spinraza was designed to treat the underlying defect in SMA, which meant it could be effective at slowing, stopping or perhaps reversing the symptoms.
The drug is currently undergoing registration with Medsafe in New Zealand.
Families of Spinal Muscular Atrophy NZ - FSMA NZ
Postal address: PO Box 4152, Christchurch
Phone: 021 609 870