55% of neuromuscular patient groups report representing patient interests “regularly” or “sometimes”, with 35% participating in Health Technology Assessment (HTA) and reimbursement reviews. Their leading activity is facilitating equitable access to healthcare (16%), followed by improving access to rare and ultra-rare therapies (14%).
55% of respondents from Neuromuscular diseases ‘Regularly’ or ‘Sometimes’ represent patient interests to drug regulators
Patient groups representing each Neuromuscular diseases sub-type
74 patient groups across 6 sub-types
interactions
Representation of patients with SMA [Spinal Muscular Atrophy] at the CONITEC [Comissão Nacional de Incorporação de Tecnologias no SUS – National Committee for Health Technology Incorporation] meeting held in December 2025; submission of responses and recommendations to CONITEC's preliminary reports on the reimbursement of SMA treatments; and mobilisation of public participation in open consultations.
Representação de pacientes com AME na reunião da Conitec ocorrida em dezembro de 2025; respostas e recomendações aos relatórios preliminares da Conitec sobre incorporação de medicamentos para AME; mobilização popular para consultas públicas.
National neuromuscular-conditions patient group, Brazil
interactions
We have testified at FDA [Food and Drug Administration] Adcomms [Advisory Committees] and have helped organise Patient-Focused Drug Development meetings.
We have testified at FDA Adcomms and have helped organize Patient-Focused Drug Development meetings; we also regularly submit comments in response to FDA RFIs.
National neuromuscular-conditions patient group, USA
actions & advocacy
Heavily advocated for access to treatment and then for SMA [Spinal Muscular Atrophy] to be added to the Newborn Screening Panel.
Heavily advocated for access to treatment and then for SMA to be added to the Newborn Screening Panel. Still advocate for adult access, but there has been less opportunity. I have personally also advocated for better access overall to medicines, as it always felt wrong to try and compete for standard of care treatments.
National neuromuscular-conditions patient group, New Zealand
actions & advocacy
Pursuing legal action to secure access to Qalsody (tofersen) — a treatment that has received FDA [Food and Drug Administration] and EMA [European Medicines Agency] approval — for Familial ALS [Amyotrophic Lateral Sclerosis] associated with the SOD1 mutation.
SOD1 mutasyonlu Ailesel ALS hastalığı için FDA, EMA onayı almış Tofersen (Qalsody) ilacına erişim konusunda hukuksal mücadele.
National neuromuscular-conditions patient group, Turkey
The percentages below highlight how the 37 patient groups interact with regulators and advocate for patient interests. As respondents may be represented in more than one area, percentages may not total 100%.
interactions
26 mentions
13 mentions
10 mentions
1 mention
1 mention
1 mention
4 mentions
2 mentions
1 mention
1 mention
3 mentions
2 mentions
1 mention
2 mentions
2 mentions
1 mention
1 mention
actions & advocacy
11 mentions
6 mentions
5 mentions
5 mentions
3 mentions
1 mention
1 mention
3 mentions
2 mentions
1 mention
3 mentions
2 mentions
1 mention
2 mentions
2 mentions
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Our research covers 2,262 patient groups representing 100 countries and 45 therapy areas. The sample report shows you the depth of analysis for a single country or therapy area. To access any other reports, please get in touch.
Visit patient-view.com to gather more information or email: Alex Wyke, alexwyke@patient-view.com
