| Biogen, Inc. · A Phase 1 Multiple-Ascending-Dose Study To Assess the Safety,Tolerability, and Pharmacokinetics of Biib105 Administered Intrathecally To Adultswith Amyotrophic Lateral Sclerosis With Or Without Poly-Cag Expansion in Theataxin-2 Gene · 2024 | Their institution | $1,418,668 |
| AveXis · Phase I, Open-Label, Dose Comparison Study of Avxs-101 for Sitting But Non-Ambulatory Patients With Spinal Muscular Atrophy · 2019 | Their institution | $831,330 |
| Biogen, Inc. · A Study To Assess the Safety, Tolerability, and Pharmacokinetics of Biib105 in Participants With Amyotrophic Lateral Sclerosis With Or Without Poly-Cytosine-Adenine-Guanine (Cag) Expansion in the Ataxin-2 Gene · 2021 | Their institution | $499,178 |
| GENZYME CORPORATION · A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Neogaa (Gz402666) and Alglucosidase Alfa in Treatment-Naive P · 2019 | Their institution | $476,035 |
| Novartis Gene Therapies, Inc. (fka AveXis, Inc.) · Phase I, Open-Label, Dose Comparison Study of Avxs-101 for Sitting But Non-Ambulatory Patients With Spinal Muscular Atrophy · 2020 | Their institution | $431,292 |
| Biogen, Inc. · A Study for Participants With Spinal Muscular Atrophy (Sma) Who Previously Participated in Nusinersen (Isis 396443) Investigational Studies. · 2021 | Their institution | $397,444 |
| Hoffmann-La Roche Limited · Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy · 2020 | Their institution | $375,633 |
| GENZYME CORPORATION · A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Neogaa (Gz402666) and Alglucosidase Alfa in Treatment-Naive Patients With Late-Onset Pompe Disease · 2020 | Their institution | $364,599 |
| Biogen, Inc. · A Study To Assess the Safety, Tolerability, and Pharmacokinetics of Biib078 in Adults With C9orf72-Associated Amyotrophic Lateral Sclerosis · 2020 | Their institution | $349,940 |
| Sarepta Therapeutics, Inc. · 2022 | Their institution | $328,910 |
| Biogen, Inc. · An Open-Label Extension Study for Patients With Spinal Muscular Atrophywho Previously Participated in Investigational Studies of Isis 396443 · 2022 | Their institution | $315,364 |
| GENZYME CORPORATION · A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Neogaa (Gz402666) and Alglucosidase Alfa in Treatment-Naive Patients With Late-Onset Pompe Disease · 2021 | Their institution | $313,141 |
| Biogen, Inc. · An Extension Study To Assess the Long-Term Safety, Tolerability,Pharmacokinetics, and Effect on Disease Progression of Biib078 Administered Topreviously Treated Adults With C9orf72-Associated Amyotrophic Lateral Sclerosis · 2022 | Their institution | $293,088 |
| Sarepta Therapeutics, Inc. · 2019 | Their institution | $284,649 |
| Biogen, Inc. · A Long-Term Extension Study of Nusinersen (Biib058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in An Investigational Study With Nusinersen. · 2023 | Their institution | $280,981 |
| F. Hoffmann-La Roche AG · Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy · 2023 | Their institution | $277,339 |
| Sarepta Therapeutics, Inc. · 2023 | Their institution | $272,496 |
| Biogen, Inc. · A Study for Participants With Spinal Muscular Atrophy (Sma) Who Previously Participated in Nusinersen (Isis 396443) Investigational Studies. · 2019 | Their institution | $261,591 |
| Biogen, Inc. · A Long-Term Extension Study of Nusinersen (Biib058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in An Investigational Study With Nusinersen. · 2024 | Their institution | $256,339 |
| Biogen, Inc. · Escalating Dose and Randomized, Controlled Study of Nusinersen (Biib058) in Participants With Spinal Muscular Atrophy · 2022 | Their institution | $233,325 |
| Biogen, Inc. · Study To Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of Biib078 Administered To Previously Treated Adults C9orf72-Associated Amyotrophic Lateral Sclerosis (Als) · 2021 | Their institution | $232,009 |
| Sarepta Therapeutics, Inc. · 2024 | Their institution | $188,007 |
| Biogen, Inc. · Study of Nusinersen (Biib058) in Participants With Spinal Muscular Atrophy · 2020 | Their institution | $172,940 |
| Hoffmann-La Roche Limited · Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy · 2024 | Their institution | $169,657 |
| Biogen, Inc. · A Long-Term Extension Study of Nusinersen (Biib058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in An Investigational Study With Nusinersen. · 2022 | Their institution | $161,427 |
| Biogen, Inc. · A Study To Assess the Safety, Tolerability, and Pharmacokinetics of Biib078 in Adults With C9orf72-Associated Amyotrophic Lateral Sclerosis · 2019 | Their institution | $158,045 |
| Biogen, Inc. · A Study for Participants With Spinal Muscular Atrophy (Sma) Who Previously Participated in Nusinersen (Isis 396443) Investigational Studies. · 2020 | Their institution | $152,424 |
| Biogen, Inc. · Nusinersen in Adults With Spinal Muscular Atrophy: An Open Label Study To Assess Efficacy and Exploratory Endpoints · 2021 | Their institution | $148,875 |
| Hoffmann-La Roche Limited · Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy · 2021 | Their institution | $134,148 |
| Biogen, Inc. · Study of Nusinersen (Biib058) in Participants With Spinal Muscular Atrophy · 2021 | Their institution | $127,583 |
| Hoffmann-La Roche Limited · Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy · 2019 | Their institution | $117,549 |
| Biogen, Inc. · Exploring Cerebrospinal Fluid Proteomic and Transcriptomic Changes in Sma After Spinraza Treatment · 2023 | Their institution | $116,947 |
| Novartis Pharmaceuticals Corporation · A Long-Term Follow-Up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving Avxs-101 · 2024 | Their institution | $116,496 |
| Biogen, Inc. · An Open-Label Extension Study for Patients With Spinal Muscular Atrophywho Previously Participated in Investigational Studies of Isis 396443 · 2024 | Their institution | $115,704 |
| Biogen, Inc. · A Long-Term Extension Study of Nusinersen (Biib058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in An Investigational Study With Nusinersen. · 2025 | Their institution | $114,028 |
| Novartis Gene Therapies, Inc. (fka AveXis, Inc.) · Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One Or Two Smn2 Copies Delivering Avxs-101 By Intravenous Infusion · 2020 | Their institution | $107,448 |
| Roche Products Limited · Ascending Dose Study in Boys With DMD · 2020 | Their institution | $107,394 |
| Sarepta Therapeutics, Inc. · Exondys 51 · 2020 | Their institution | $107,206 |
| Biogen, Inc. · Exploring Cerebrospinal Fluid Proteomic and Transcriptomic Changes in Sma After Spinraza Treatment · 2020 | Their institution | $102,329 |
| Roche Products Limited · Study To Assess Efficacy Safety and Tolerability in Ambulatory Boys With DMD · 2019 | Their institution | $100,831 |
| Biogen, Inc. · A Phase 4 Study of Nusinersen (Biib058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec · 2022 | Their institution | $98,224 |
| Biogen, Inc. · Study To Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of Biib078 Administered To Previously Treated Adults C9orf72-Associated Amyotrophic Lateral Sclerosis (Als) · 2020 | Their institution | $96,151 |
| Hoffmann-La Roche Limited · Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy · 2022 | Their institution | $93,913 |
| Biogen, Inc. · A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec · 2021 | Their institution | $93,517 |
| Biogen, Inc. · Extension Study of Nusinersen (Biib058) in Participants With Spinal Muscular Atrophy Who Previously Participated in A Study With Nusinersen · 2021 | Their institution | $91,695 |
| Biogen, Inc. · Nusinersen in Adults With Spinal Muscular Atrophy: An Open Label Study To Assess Efficacy and Exploratory Endpoints · 2025 | Their institution | $82,609 |
| Biogen, Inc. · A Study To Assess the Safety, Tolerability, and Pharmacokinetics of Biib078 in Adults With C9orf72-Associated Amyotrophic Lateral Sclerosis · 2021 | Their institution | $80,521 |
| Novartis Gene Therapies, Inc. (fka AveXis, Inc.) · Phase I, Open-Label, Dose Comparison Study of Avxs-101 for Sitting But Non-Ambulatory Patients With Spinal Muscular Atrophy · 2021 | Their institution | $77,101 |
| Sarepta Therapeutics, Inc. · Amondys 45 · 2024 | Their institution | $75,680 |
| Biogen, Inc. · An Open-Label Extension Study for Patients With Spinal Muscular Atrophywho Previously Participated in Investigational Studies of Isis 396443 · 2023 | Their institution | $73,848 |
| Biogen, Inc. · Exploring Cerebrospinal Fluid Proteomic and Transcriptomic Changes in Sma After Spinraza Treatment · 2022 | Their institution | $73,092 |
| Sarepta Therapeutics, Inc. · Vyondys 53 · 2020 | Their institution | $70,517 |
| Roche Products Limited · Study To Assess Efficacy Safety and Tolerability in Ambulatory Boys With DMD · 2020 | Their institution | $70,480 |
| Biogen, Inc. · A Phase 4 Study of Nusinersen (Biib058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec · 2023 | Their institution | $66,045 |
| Hoffmann-La Roche Limited · Ro7034067 in Infants With Type 1 Spinal Muscular Atrophy · 2019 | Their institution | $65,565 |
| Hoffmann-La Roche Limited · Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients · 2023 | Their institution | $65,481 |
| Hoffmann-La Roche Limited · Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients · 2024 | Their institution | $65,265 |
| Hoffmann-La Roche Limited · Ascending Dose Study in Boys With DMD · 2019 | Their institution | $63,149 |
| Novartis Gene Therapies, Inc. (fka AveXis, Inc.) · Phase I, Open-Label, Dose Comparison Study of Avxs-101 for Sitting But Non-Ambulatory Patients With Spinal Muscular Atrophy · 2022 | Their institution | $60,714 |
| Novartis Pharmaceuticals Corporation · Assessing Pediatric Endpoints in Dm1 (Aspire-Dm1) · 2024 | Their institution | $56,628 |
| Biogen, Inc. · A Phase 3b Study To Evaluate Higher Dose Nusinersen (Biib058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam · 2025 | Their institution | $55,572 |
| Biogen, Inc. · Nusinersen in Adults With Spinal Muscular Atrophy: An Open Label Study To Assess Efficacy and Exploratory Endpoints · 2023 | Their institution | $55,073 |
| Novartis Pharmaceuticals Corporation · A Long-Term Follow-Up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving Avxs-101 · 2025 | Their institution | $53,898 |
| Biogen, Inc. · A Phase 1 Multiple-Ascending-Dose Study To Assess the Safety,Tolerability, and Pharmacokinetics of Biib105 Administered Intrathecally To Adultswith Amyotrophic Lateral Sclerosis With Or Without Poly-Cag Expansion in Theataxin-2 Gene · 2023 | Their institution | $51,272 |
| Biogen, Inc. · A Phase 3b Study To Evaluate Higher Dose Nusinersen (Biib058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam · 2024 | Their institution | $50,195 |
| Hoffmann-La Roche Limited · Study To Assess Efficacy Safety and Tolerability in Ambulatory Boys With DMD · 2019 | Their institution | $48,011 |
| Novartis Gene Therapies, Inc. (fka AveXis, Inc.) · A Long-Term Follow-Up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving Avxs-101 · 2022 | Their institution | $47,581 |
| Roche Products Limited · Ascending Dose Study in Boys With DMD · 2019 | Their institution | $46,997 |
| Hoffmann-La Roche Limited · Ro7034067 in Infants With Type 1 Spinal Muscular Atrophy · 2023 | Their institution | $45,936 |
| F. Hoffmann-La Roche AG · Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients · 2023 | Their institution | $45,317 |
| Hoffmann-La Roche Limited · Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients · 2019 | Their institution | $38,510 |
| Sarepta Therapeutics, Inc. · Amondys 45 · 2021 | Their institution | $38,064 |
| Hoffmann-La Roche Limited · Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy · 2025 | Their institution | $36,695 |
| Biogen, Inc. · Neuropathologic and Autoradiographic Study of Tauopathy in Myotonic Dystrophy (Dm1) · 2021 | Their institution | $33,975 |
| Sarepta Therapeutics, Inc. · 2021 | Their institution | $32,905 |
| Ionis Pharmaceuticals, Inc. · Ion363cs1 · 2025 | Their institution | $32,837 |
| Hoffmann-La Roche Limited · Ro7034067 in Infants With Type 1 Spinal Muscular Atrophy · 2022 | Their institution | $32,051 |
| Novartis Gene Therapies, Inc. (fka AveXis, Inc.) · A Global Study of A Single, One-Time Dose of Avxs-101 Delivered To Infants With Genetically Diagnosed and Pre-Symptomatic Spinal Muscular Atrophy With Multiple Copies of Smn2 · 2020 | Their institution | $31,484 |
| Biogen, Inc. · A Phase 1 Multiple-Ascending-Dose Study To Assess the Safety,Tolerability, and Pharmacokinetics of Biib105 Administered Intrathecally To Adultswith Amyotrophic Lateral Sclerosis With Or Without Poly-Cag Expansion in Theataxin-2 Gene · 2022 | Their institution | $31,334 |
| PTC Therapeutics, Inc. · A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension · 2020 | Their institution | $31,034 |
| Ionis Pharmaceuticals, Inc. · A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study To Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered Ion373 in Patients With Alexander Disease · 2025 | Their institution | $30,726 |
| GENZYME CORPORATION · A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Neogaa (Gz402666) and Alglucosidase Alfa in Treatment-Naive Patients With Late-Onset Pompe Disease · 2023 | Their institution | $30,287 |
| PTC Therapeutics, Inc. · A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension · 2019 | Their institution | $29,733 |
| Biogen, Inc. · Escalating Dose and Randomized, Controlled Study of Nusinersen (Biib058) in Participants With Spinal Muscular Atrophy · 2023 | Their institution | $28,677 |
| Biogen, Inc. · Nusinersen in Adults With Spinal Muscular Atrophy: An Open Label Study To Assess Efficacy and Exploratory Endpoints · 2024 | Their institution | $27,536 |
| Biogen, Inc. · A Phase 3b Study To Evaluate Higher Dose Nusinersen (Biib058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam · 2023 | Their institution | $27,294 |
| Hoffmann-La Roche Limited · Clinical Trial To Evaluate the Efficacy, Safety, and Tolerability of Ro7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy · 2019 | Their institution | $26,867 |
| F. Hoffmann-La Roche AG · Ro7034067 in Infants With Type 1 Spinal Muscular Atrophy · 2023 | Their institution | $25,527 |
| Hoffmann-La Roche Limited · Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients · 2020 | Their institution | $24,773 |
| Sarepta Therapeutics, Inc. · 2025 | Their institution | $24,479 |
| Hoffmann-La Roche Limited · Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy · 2023 | Their institution | $23,929 |
| Novartis Gene Therapies, Inc. (fka AveXis, Inc.) · A Long-Term Follow-Up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving Avxs-101 · 2023 | Their institution | $23,222 |
| Novartis Gene Therapies, Inc. (fka AveXis, Inc.) · A Long-Term Follow-Up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving Avxs-101 · 2021 | Their institution | $23,021 |
| Hoffmann-La Roche Limited · Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients · 2022 | Their institution | $22,482 |
| Hoffmann-La Roche Limited · Ro7034067 in Infants With Type 1 Spinal Muscular Atrophy · 2020 | Their institution | $21,910 |
| PTC Therapeutics, Inc. · An Open-Label, Safety Study for Previously Treated Ataluren Patients With Nonsense Mutation Dystrophinopathy · 2021 | Their institution | $21,342 |
| Sarepta Therapeutics, Inc. · Chart Review · 2019 | Their institution | $21,175 |
| Biogen, Inc. · An Open Label, Single Cohort Study To Assess the Pharmacokinetic Profile of Nusinersen (Biib058) Administered Via the Thecaflex Drx System (Pierre-Pk) · 2025 | Their institution | $20,710 |
| Biogen, Inc. · A Phase 1 Multiple-Ascending-Dose Study To Assess the Safety, Tolerability, and Pharmacokinetics of Biib078 Administered Intrathecally To Adults With C9orf72-Associated Amyotrophic Lateral Sclerosis · 2022 | Their institution | $20,188 |
| Genentech, Inc. · Risdiplam Post Gene Therapy Study Plateau Symptoms Study 2 · 2024 | Their institution | $19,763 |
| Hoffmann-La Roche Limited · Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients · 2021 | Their institution | $19,489 |
| Novartis Gene Therapies, Inc. (fka AveXis, Inc.) · A Long-Term Follow-Up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving Avxs-101 · 2020 | Their institution | $18,072 |
| PTC Therapeutics, Inc. · An Open-Label, Safety Study for Ataluren (Ptc124) Patients With Nonsense Mutation Dystrophinopathy · 2023 | Their institution | $17,682 |
| GENZYME CORPORATION · A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Neogaa (Gz402666) and Alglucosidase Alfa in Treatment-Naive Patients With Late-Onset Pompe Disease · 2022 | Their institution | $16,893 |
| Genentech, Inc. · Risdiplam Post Gene Therapy Study Plateau Symptoms Study 2 · 2025 | Their institution | $16,790 |
| VERTEX PHARMACEUTICALS INCORPORATED · A Feasibility Study To Assess the Use of A Digital Device in Subjects With Myotonic Dystrophy Type 1 and Healthy Volunteers · 2023 | Their institution | $16,140 |
| PTC Therapeutics, Inc. · An Open-Label, Safety Study for Ataluren (Ptc124) Patients With Nonsense Mutation Dystrophinopathy · 2025 | Their institution | $14,114 |
| PTC Therapeutics, Inc. · A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension · 2021 | Their institution | $13,913 |
| Hoffmann-La Roche Limited · A Study To Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Sma Participants Sunfish · 2019 | Their institution | $12,078 |
| Hoffmann-La Roche Limited · Ro7034067 in Infants With Type 1 Spinal Muscular Atrophy · 2024 | Their institution | $11,332 |
| PTC Therapeutics, Inc. · An Open-Label, Safety Study for Ataluren (Ptc124) Patients With Nonsense Mutation Dystrophinopathy · 2024 | Their institution | $10,643 |
| F. Hoffmann-La Roche AG · A Two Part Seamless, Open-Label, Multicenter Study To Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Ro7034067 in Infants With Type 1 Spinal Muscular Atrophy · 2020 | This clinician | $9,382 |
| PTC Therapeutics, Inc. · A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension · 2022 | Their institution | $9,360 |
| PTC Therapeutics, Inc. · An Open-Label, Safety Study for Ataluren (Ptc124) Patients With Nonsense Mutation Dystrophinopathy · 2022 | Their institution | $8,172 |
| Biogen, Inc. · Escalating Dose and Randomized, Controlled Study of Nusinersen (Biib058) in Participants With Spinal Muscular Atrophy · 2025 | Their institution | $7,805 |
| Biogen, Inc. · A Phase 4 Study of Nusinersen (Biib058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec · 2024 | Their institution | $7,486 |
| Biogen, Inc. · A Phase 4 Study of Nusinersen (Biib058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec · 2025 | Their institution | $6,195 |
| Biogen, Inc. · Escalating Dose and Randomized, Controlled Study of Nusinersen (Biib058) in Participants With Spinal Muscular Atrophy · 2024 | Their institution | $5,035 |
| PTC Therapeutics, Inc. · A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension · 2025 | Their institution | $5,000 |
| F. Hoffmann-La Roche AG · A Two-Part Seamless, Multi-Center Randomized, Placebo-Controlled, Doubleblind Study To Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients. · 2020 | This clinician | $4,834 |
| PTC Therapeutics, Inc. · A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension · 2023 | Their institution | $4,159 |
| SANOFI US SERVICES INC. · Pompe Disease Registry · 2023 | Their institution | $3,475 |
| Sarepta Therapeutics, Inc. · Amondys 45 · 2022 | Their institution | $3,186 |
| Biogen, Inc. · An Extension Study To Assess the Long-Term Safety, Tolerability,Pharmacokinetics, and Effect on Disease Progression of Biib078 Administered Topreviously Treated Adults With C9orf72-Associated Amyotrophic Lateral Sclerosis · 2023 | Their institution | $3,129 |
| F. Hoffmann-La Roche AG · Study of Rg7916 in Pre Symptomatic Sma Patients · 2023 | This clinician | $2,749 |
| Sarepta Therapeutics, Inc. · Amondys 45 · 2023 | Their institution | $2,560 |
| Hoffmann-La Roche Limited · Ro7034067 in Infants With Type 1 Spinal Muscular Atrophy · 2021 | Their institution | $2,281 |
| GENZYME CORPORATION · A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Neogaa (Gz402666) and Alglucosidase Alfa in Treatment-Naive Patients With Late-Onset Pompe Disease · 2021 | This clinician | $2,000 |
| Biogen, Inc. · Nusinersen in Adults With Spinal Muscular Atrophy: An Open Label Study To Assess Efficacy and Exploratory Endpoints · 2022 | Their institution | $1,972 |
| F. Hoffmann-La Roche AG · An Open-Label Study To Investigate the Safety, Tolerability, and Pharmacokineticspharmacodynamics of Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy · 2020 | This clinician | $1,802 |
| GENZYME CORPORATION · A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Neogaa (Gz402666) and Alglucosidase Alfa in Treatment-Naive Patients With Late-Onset Pompe Disease · 2022 | This clinician | $1,750 |
| F. Hoffmann-La Roche AG · Baseline Demographics and Clinical Characteristics of Risdiplam-Treated Individuals With Sma From the Pediatric Neuromuscular Clinical Research (Pncr) Registry · 2025 | This clinician | $1,681 |
| F. Hoffmann-La Roche AG · An Open-Label Study To Investigate the Safety, Tolerability, and Pharmacokineticspharmacodynamics of Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy, A Two-Part Seamless, Multi-Center Randomized, Placebo-Controlled, Doubleblind Study To Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients., A Two Part Seamless, Open-Label, Multicenter Study To Investigate the Safety, in · 2020 | This clinician | $1,539 |
| GENZYME CORPORATION · A Phase 4, Open Label, Prospective Study in Patients With Pompe Disease To Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000l Scale · 2019 | This clinician | $1,352 |
| F. Hoffmann-La Roche AG · An Open-Label Study To Investigate the Safety, Tolerability, and Pharmacokineticspharmacodynamics of Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy, A Two-Part Seamless, Multi-Center Randomized, Placebo-Controlled, Doubleblind Study To Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients., A Two Part Seamless, Open-Label, Multicenter Study To Investigate the Safety, Tolerabilit · 2021 | This clinician | $1,292 |
| F. Hoffmann-La Roche AG · A Two-Part Seamless, Multi-Center Randomized, Placebo-Controlled, Doubleblind Study To Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients. · 2021 | This clinician | $1,019 |
| F. Hoffmann-La Roche AG · A Two Part Seamless, Open-Label, Multicenter Study To Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Ro7034067 in Infants With Type 1 Spinal Muscular Atrophy · 2021 | This clinician | $1,009 |
| F. Hoffmann-La Roche AG · Ro7034067 in Infants With Type 1 Spinal Muscular Atrophy · 2024 | This clinician | $989 |
| Novartis Gene Therapies, Inc. (fka Avexis, Inc.) · Research Medical Writing and Publication Support · 2024 | This clinician | $963 |
| GENZYME CORPORATION · A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Neogaa (Gz402666) and Alglucosidase Alfa in Treatment-Naive Patients With Late-Onset Pompe Disease · 2023 | This clinician | $750 |
| GENZYME CORPORATION · A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Neogaa (Gz402666) and Alglucosidase Alfa in Treatment Naive Patients With Late Onset Pompe Disease · 2021 | This clinician | $750 |
| Novartis Pharmaceuticals Corporation · A Long-Term Follow-Up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving Avxs-101 · 2022 | Their institution | $737 |
| F. Hoffmann-La Roche AG · Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients · 2025 | This clinician | $708 |
| GENZYME CORPORATION · A Phase 4, Open Label, Prospective Study in Patients With Pompe Disease To Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000l Scale · 2020 | This clinician | $524 |
| F. Hoffmann-La Roche AG · Safety Update: Risdiplam Clinical Trial Program for Spinal Muscular Atrophy (Sma) · 2025 | This clinician | $504 |
| F. Hoffmann-La Roche AG · Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy · 2025 | This clinician | $501 |
| F. Hoffmann-La Roche AG · Firefish Parts 1 and 2: 5-Year Efficacy and Safety of Risdiplam in Type 1 Sma · 2025 | This clinician | $459 |
| F. Hoffmann-La Roche AG · Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy (DMD) · 2023 | This clinician | $385 |
| F. Hoffmann-La Roche AG · Safety Update: Risdiplam Clinical Trial Program for Sma · 2023 | This clinician | $354 |
| GENZYME CORPORATION · A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Neogaa (Gz402666) and Alglucosidase Alfa in Treatment-Nave · 2020 | This clinician | $340 |
| Biogen, Inc. · A Phase 3b Study To Evaluate Higher Dose Nusinersen (Biib058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam · 2022 | Their institution | $320 |
| F. Hoffmann-La Roche AG · A Gene Delivery Study To Evaluate the Safety of and Expression From Srp-9001 in Duchenne Muscular Dystrophy (DMD) (Endeavor) · 2024 | This clinician | $307 |
| F. Hoffmann-La Roche AG · Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients · 2020 | This clinician | $303 |
| Novartis Pharmaceuticals Corporation · Restore Registry · 2025 | This clinician | $249 |
| F. Hoffmann-La Roche AG · Study of Rg7916 in Pre Symptomatic Sma Patients, An Open-Label Study To Investigate the Safety, Tolerability, and Pharmacokineticspharmacodynamics of Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy, A Two-Part Seamless, Multi-Center Randomized, Placebo-Controlled, Doubleblind Study To Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Ro7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients., A Two Part Seamless, Open-Label, Multice · 2021 | This clinician | $220 |
| GENZYME CORPORATION · A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Neogaa (Gz402666) and Alglucosidase Alfa in Treatment-Naive Patients With Late-Onset Pompe Disease · 2020 | This clinician | $217 |
| Biogen, Inc. · Study of Nusinersen (Biib058) in Participants With Spinal Muscular Atrophy · 2020 | This clinician | $136 |
| F. Hoffmann-La Roche AG · An Open-Label Study To Investigate the Safety, Tolerability, and Pharmacokineticspharmacodynamics of Ro7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy · 2021 | This clinician | $71 |
| Novartis Pharmaceuticals Corporation · A Long-Term Follow-Up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving Avxs-101 · 2023 | Their institution | $70 |