ACE-031
Dr. Diana Escolar is back and her trial ACE-031 will be underway soon - and there will be five sites in the US for the next trial. FDA loves the trial. Cannot wait for it. It may beat Prosensa (GSK) trial here.
Yesterday, she began seeing patients again, only four per month so she can devote the rest of her time to research. SHe is completely excited about this trial.
Replies to This Discussion
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Permalink Reply by Kristi Koop on
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What other information do you have on the ACE-031 trial? Ideas on where the 5 sites might be?
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Permalink Reply by Karen flor on
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Hi! I have read many things about both the Prosena {exon skipping 51} and ACE-031 but need help!! Comparing the two, what are the advantages of one over the other or are there???? From what I understand ACE-031 can be used for a wider scope of boys than exon skipping 51. However, my son fits into the exon skipping 51 group and I'm hoping to get him into a trial, but would like to know if I'm barking up the wrong tree and the ACE-031 might be a better treatment. Any thoughts???
Karen
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Permalink Reply by irishgirl on
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That is all the information I have at present. The trial is already underway in Canada and the five US sites will be determined. I wish I had more to offer. Waiting is so very difficult.
Kristi Koop said:What other information do you have on the ACE-031 trial? Ideas on where the 5 sites might be?
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Hi Karen: Dr. Escolar says that one is really no better than the other. They do the same thing, just different compounds. At present - it's which ever gets here first. Keep in mind though that generally if you are included in one trial, you are generally excluded from another for a least a year. They do not want traces of another drug in the body while instilling a new one. Would throw results off. None of these are cures, but treatments in a time when there are none. We are fairly lucky to have three treatments coming soon down the line. Escolar did say that the ACE-031 would be for ambulatory boys.
How old is your son - how is his mobility?
Karen flor said:Hi! I have read many things about both the Prosena {exon skipping 51} and ACE-031 but need help!! Comparing the two, what are the advantages of one over the other or are there???? From what I understand ACE-031 can be used for a wider scope of boys than exon skipping 51. However, my son fits into the exon skipping 51 group and I'm hoping to get him into a trial, but would like to know if I'm barking up the wrong tree and the ACE-031 might be a better treatment. Any thoughts???
Karen
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Permalink Reply by Dana Edwards on
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Hi, I look at this all the time and I am not sure but I think this will help a lot of us. My son has a large in-frame deletion and this doesn't look like it has anything to do with exon skipping. I may be wrong but I am keeping my fingers crossed. I just went to their website and this was just updated as of June 3rd. If you understand it better than me please explain it. I get crazy with this. We all need our MIRACLE!!!!
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
This study is currently recruiting participants.
Verified by Acceleron Pharma, Inc., June 2010
First Received: April 2, 2010 Last Updated: June 1, 2010 History of Changes
Sponsor: Acceleron Pharma, Inc.
Information provided by: Acceleron Pharma, Inc.
ClinicalTrials.gov Identifier: NCT01099761
Purpose
The purpose of this study is to determine if ACE-031 is safe and well-tolerated in children with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for designing future studies.
Condition Intervention Phase
Duchenne Muscular Dystrophy
Biological: ACE-031
Other: Placebo
Phase II
Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
Resource links provided by NLM:
Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy
MedlinePlus related topics: Muscular Dystrophy
U.S. FDA Resources
Further study details as provided by Acceleron Pharma, Inc.:
Primary Outcome Measures:
Safety and tolerability by monitoring adverse events, clinical laboratory tests, electrocardiogram (ECG), echocardiogram (ECHO), physical examinations, vital signs, and anti-drug antibodies [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
Percent change from baseline in total body lean mass by DXA [ Time Frame: 4 months ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
Change from baseline in timed function tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Change from baseline in muscle strength tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Change from baseline in pulmonary function tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Estimated Enrollment: 76
Study Start Date: April 2010
Estimated Study Completion Date: February 2012
Estimated Primary Completion Date: February 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
ACE-031: Experimental
Patients assigned to 1 of 4 possible dosing groups Biological: ACE-031
Subcutaneous dose of ACE-031 every 4 weeks for 12 weeks, for a total of 4 doses.
Placebo: Placebo Comparator Other: Placebo
Subcutaneous dose of placebo every 4 weeks for 12 weeks, for a total of 4 doses.
Eligibility
Ages Eligible for Study: 4 Years and older
Genders Eligible for Study: Male
Accepts Healthy Volunteers: No
Criteria
Inclusion Criteria:
Diagnosis of DMD confirmed
Ambulant
Corticosteroid therapy for at least one year prior to study day 1 and on a stable dose and schedule for at least 6 months prior to study day 1
Evidence of muscle weakness by clinical assessment
Exclusion Criteria:
Any previous treatment with another investigational product within 6 months prior to study day 1
Any clinically significant cardiac, endocrine, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, and/or other major disease that is not related to DMD
Inability to perform a whole body dual x-ray absorptiometry (DXA) scan
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01099761
Contacts
Contact: Rhiannon Taranik 519-685-8441 Rhiannon.Taranik@lhsc.on.ca
Contact: Trial Manager clinicaltrials@acceleronpharma.com
Locations
Canada, Alberta
Acceleron Investigative Site Recruiting
Calgary, Alberta, Canada
Canada, Ontario
Acceleron Investigative Site Recruiting
London, Ontario, Canada
Sponsors and Collaborators
Acceleron Pharma, Inc.
More Information
No publications provided
Responsible Party: Acceleron Pharma, Inc. ( Kenneth M. Attie, MD, Medical Monitor )
ClinicalTrials.gov Identifier: NCT01099761 History of Changes
Other Study ID Numbers: A031-03
Study First Received: April 2, 2010
Last Updated: June 1, 2010
Health Authority: Canada: Health Canada
Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Diseases
Genetic Diseases, Inborn
Neuromuscular Diseases
Musculoskeletal Diseases
Muscular Disorders, Atrophic
Muscular Dystrophy, Duchenne
Nervous System Diseases
Genetic Diseases, X-Linked
ClinicalTrials.gov processed this record on June 03, 2010
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Dana: What deletions does your son have? The exons are like a puzzle that fit together. My son is missing exons 48, 49 and 50. Exon 47 doesn't fit into 51, but if exon 51 is skipped, exon 47 fits perfectly into exon 52. Our son, fits the bill here to a tee.
Depending on what your son is missing will determine if exon 51 skipping is appropriate for him. Exon 51 skipping was the first because it has the possibilities of helping the most children with DMD - it's like 12 or 13% of those with DMD.
The ACE-031 information which you got from clinicaltrials.gov, right shows the trial is recruiting, but only in Canada at present, but according to Dr. Escolar, five site will be opening up in the US - extending this trial. It is hoped that this will do the same as Prosensa's (GSK) upcoming trial and AVI's as well. But, if these drugs continue to perform as well as they appear to be, the entire face of DMD will be changed for the better. None of these are cures, but with these drugs and others - like a cocktail, we may be able to manage the DMD in the way AIDS is now managed. Not cured, but managed.
You need find out if your son is a fit for these trials/drugs. Also, if your son has an in frame deletion, he may as well have Becker and these trials would not be applicable. My son is out of frame with the proper deletions to be a perfect candidate for any of these three drugs - they make and out of frame in frame.
I hope this helps - somewhat. Never loose faith in managed care - it really is around the corner.
Dana Edwards said:Hi, I look at this all the time and I am not sure but I think this will help a lot of us. My son has a large in-frame deletion and this doesn't look like it has anything to do with exon skipping. I may be wrong but I am keeping my fingers crossed. I just went to their website and this was just updated as of June 3rd. If you understand it better than me please explain it. I get crazy with this. We all need our MIRACLE!!!!
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
This study is currently recruiting participants.
Verified by Acceleron Pharma, Inc., June 2010
First Received: April 2, 2010 Last Updated: June 1, 2010 History of Changes
Sponsor: Acceleron Pharma, Inc.
Information provided by: Acceleron Pharma, Inc.
ClinicalTrials.gov Identifier: NCT01099761
Purpose
The purpose of this study is to determine if ACE-031 is safe and well-tolerated in children with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for designing future studies.
Condition Intervention Phase
Duchenne Muscular Dystrophy
Biological: ACE-031
Other: Placebo
Phase II
Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
Resource links provided by NLM:
Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy
MedlinePlus related topics: Muscular Dystrophy
U.S. FDA Resources
Further study details as provided by Acceleron Pharma, Inc.:
Primary Outcome Measures:
Safety and tolerability by monitoring adverse events, clinical laboratory tests, electrocardiogram (ECG), echocardiogram (ECHO), physical examinations, vital signs, and anti-drug antibodies [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
Percent change from baseline in total body lean mass by DXA [ Time Frame: 4 months ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
Change from baseline in timed function tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Change from baseline in muscle strength tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Change from baseline in pulmonary function tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Estimated Enrollment: 76
Study Start Date: April 2010
Estimated Study Completion Date: February 2012
Estimated Primary Completion Date: February 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
ACE-031: Experimental
Patients assigned to 1 of 4 possible dosing groups Biological: ACE-031
Subcutaneous dose of ACE-031 every 4 weeks for 12 weeks, for a total of 4 doses.
Placebo: Placebo Comparator Other: Placebo
Subcutaneous dose of placebo every 4 weeks for 12 weeks, for a total of 4 doses.
Eligibility
Ages Eligible for Study: 4 Years and older
Genders Eligible for Study: Male
Accepts Healthy Volunteers: No
Criteria
Inclusion Criteria:
Diagnosis of DMD confirmed
Ambulant
Corticosteroid therapy for at least one year prior to study day 1 and on a stable dose and schedule for at least 6 months prior to study day 1
Evidence of muscle weakness by clinical assessment
Exclusion Criteria:
Any previous treatment with another investigational product within 6 months prior to study day 1
Any clinically significant cardiac, endocrine, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, and/or other major disease that is not related to DMD
Inability to perform a whole body dual x-ray absorptiometry (DXA) scan
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01099761
Contacts
Contact: Rhiannon Taranik 519-685-8441 Rhiannon.Taranik@lhsc.on.ca
Contact: Trial Manager clinicaltrials@acceleronpharma.com
Locations
Canada, Alberta
Acceleron Investigative Site Recruiting
Calgary, Alberta, Canada
Canada, Ontario
Acceleron Investigative Site Recruiting
London, Ontario, Canada
Sponsors and Collaborators
Acceleron Pharma, Inc.
More Information
No publications provided
Responsible Party: Acceleron Pharma, Inc. ( Kenneth M. Attie, MD, Medical Monitor )
ClinicalTrials.gov Identifier: NCT01099761 History of Changes
Other Study ID Numbers: A031-03
Study First Received: April 2, 2010
Last Updated: June 1, 2010
Health Authority: Canada: Health Canada
Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Diseases
Genetic Diseases, Inborn
Neuromuscular Diseases
Musculoskeletal Diseases
Muscular Disorders, Atrophic
Muscular Dystrophy, Duchenne
Nervous System Diseases
Genetic Diseases, X-Linked
ClinicalTrials.gov processed this record on June 03, 2010
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Hi Dana: irishgirl here again. Has your son had a muscle biopsy yet? That is really the only way to tell or to pinpoint what's he's got. My son will be 11 this November and the doctors say he is at the extreme end of the DMD bell curve. One end has the most serious cases where children show symptoms as early as 18 months. The middle is the bell where most DMD kids are - then there is the extreme other end where my son is. Appears to be the exact opposite of your son. The bx confirmed DMD, but my son's condition is far more like a Becker MD while he does have DMD. He is extremely active with running, jumping, swimming - all sorts of things. Dr. Escolar has never had a patient like Liam ever before. We drink to that every day. But this horrid disease is so strange on so many different levels with the same outcome of being 100% fatal at present. But since your son is only 4, right - it is hard to say what he will be like when he's 8 or 10 or 12. He's very young. Our son was diagnosed at 8 with his symptom being that he ran slower than his buddies.
Does your son show any symptoms at all yet? Please tell me more about him.
Noreen
Dana Edwards said:Hi, I look at this all the time and I am not sure but I think this will help a lot of us. My son has a large in-frame deletion and this doesn't look like it has anything to do with exon skipping. I may be wrong but I am keeping my fingers crossed. I just went to their website and this was just updated as of June 3rd. If you understand it better than me please explain it. I get crazy with this. We all need our MIRACLE!!!!
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
This study is currently recruiting participants.
Verified by Acceleron Pharma, Inc., June 2010
First Received: April 2, 2010 Last Updated: June 1, 2010 History of Changes
Sponsor: Acceleron Pharma, Inc.
Information provided by: Acceleron Pharma, Inc.
ClinicalTrials.gov Identifier: NCT01099761
Purpose
The purpose of this study is to determine if ACE-031 is safe and well-tolerated in children with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for designing future studies.
Condition Intervention Phase
Duchenne Muscular Dystrophy
Biological: ACE-031
Other: Placebo
Phase II
Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
Resource links provided by NLM:
Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy
MedlinePlus related topics: Muscular Dystrophy
U.S. FDA Resources
Further study details as provided by Acceleron Pharma, Inc.:
Primary Outcome Measures:
Safety and tolerability by monitoring adverse events, clinical laboratory tests, electrocardiogram (ECG), echocardiogram (ECHO), physical examinations, vital signs, and anti-drug antibodies [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
Percent change from baseline in total body lean mass by DXA [ Time Frame: 4 months ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
Change from baseline in timed function tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Change from baseline in muscle strength tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Change from baseline in pulmonary function tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Estimated Enrollment: 76
Study Start Date: April 2010
Estimated Study Completion Date: February 2012
Estimated Primary Completion Date: February 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
ACE-031: Experimental
Patients assigned to 1 of 4 possible dosing groups Biological: ACE-031
Subcutaneous dose of ACE-031 every 4 weeks for 12 weeks, for a total of 4 doses.
Placebo: Placebo Comparator Other: Placebo
Subcutaneous dose of placebo every 4 weeks for 12 weeks, for a total of 4 doses.
Eligibility
Ages Eligible for Study: 4 Years and older
Genders Eligible for Study: Male
Accepts Healthy Volunteers: No
Criteria
Inclusion Criteria:
Diagnosis of DMD confirmed
Ambulant
Corticosteroid therapy for at least one year prior to study day 1 and on a stable dose and schedule for at least 6 months prior to study day 1
Evidence of muscle weakness by clinical assessment
Exclusion Criteria:
Any previous treatment with another investigational product within 6 months prior to study day 1
Any clinically significant cardiac, endocrine, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, and/or other major disease that is not related to DMD
Inability to perform a whole body dual x-ray absorptiometry (DXA) scan
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01099761
Contacts
Contact: Rhiannon Taranik 519-685-8441 Rhiannon.Taranik@lhsc.on.ca
Contact: Trial Manager clinicaltrials@acceleronpharma.com
Locations
Canada, Alberta
Acceleron Investigative Site Recruiting
Calgary, Alberta, Canada
Canada, Ontario
Acceleron Investigative Site Recruiting
London, Ontario, Canada
Sponsors and Collaborators
Acceleron Pharma, Inc.
More Information
No publications provided
Responsible Party: Acceleron Pharma, Inc. ( Kenneth M. Attie, MD, Medical Monitor )
ClinicalTrials.gov Identifier: NCT01099761 History of Changes
Other Study ID Numbers: A031-03
Study First Received: April 2, 2010
Last Updated: June 1, 2010
Health Authority: Canada: Health Canada
Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Diseases
Genetic Diseases, Inborn
Neuromuscular Diseases
Musculoskeletal Diseases
Muscular Disorders, Atrophic
Muscular Dystrophy, Duchenne
Nervous System Diseases
Genetic Diseases, X-Linked
ClinicalTrials.gov processed this record on June 03, 2010
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Hi! Thanks for the imput...My son is 10 1/2 and thank God completely ambulatory..He has a hard time with stairs, but can still do them, walks everywhere for long periods but doesn't run much...partly because of physical limitations and the other part because of embarassment in front of his friends that he's "different"..I was aware that you can't do more than one study at a time and need time inbetween....thats why I want all the info possible to make the best decision. We are so blessed to have options coming, but with everything so new I feel a little pressure to make the right choices. I have Lucas set up to have his first visit with Dr. Wong in July, but I'm unfamilar with Dr.Escolar....where is he/she????
irishgirl said:Hi Karen: Dr. Escolar says that one is really no better than the other. They do the same thing, just different compounds. At present - it's which ever gets here first. Keep in mind though that generally if you are included in one trial, you are generally excluded from another for a least a year. They do not want traces of another drug in the body while instilling a new one. Would throw results off. None of these are cures, but treatments in a time when there are none. We are fairly lucky to have three treatments coming soon down the line. Escolar did say that the ACE-031 would be for ambulatory boys.
How old is your son - how is his mobility?
Karen flor said:Hi! I have read many things about both the Prosena {exon skipping 51} and ACE-031 but need help!! Comparing the two, what are the advantages of one over the other or are there???? From what I understand ACE-031 can be used for a wider scope of boys than exon skipping 51. However, my son fits into the exon skipping 51 group and I'm hoping to get him into a trial, but would like to know if I'm barking up the wrong tree and the ACE-031 might be a better treatment. Any thoughts???
Karen
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Permalink Reply by Michelle on
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http://www.acceleronpharma.com/content/products/ace-031.jsp
ACE-031 isn't just for DMD. It's a muscle building compound for muscle wasting diseases in general. It doesn't cure DMD or assume to. It just builds muscle. In our case, it's more of a race against time. Build muscle in the hopes of delaying the tissue degeneration.
Karen flor said:Hi! I have read many things about both the Prosena {exon skipping 51} and ACE-031 but need help!! Comparing the two, what are the advantages of one over the other or are there???? From what I understand ACE-031 can be used for a wider scope of boys than exon skipping 51. However, my son fits into the exon skipping 51 group and I'm hoping to get him into a trial, but would like to know if I'm barking up the wrong tree and the ACE-031 might be a better treatment. Any thoughts???
Karen
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Permalink Reply by Tina on
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Hi Karen. Dr. Escolar sent me this email a while back. I'm sure she wouldn't mind it if I share it with this group. Here is where you can find her. Best of luck.
Tina
Dear Families, Friends and Colleagues,
I've signed a contract with Kennedy Krieger Institute in Baltimore and will start seeing patients again on a part time basis at their new Center for Inherited Muscle Disorders. Depending on how long it takes to obtain my Maryland license, my first clinic will be once a month (on Fridays) starting probably late November or December.
Dr. Kathryn Wagner is the director of the Center, and has started seeing patients in July. At this new Muscle Clinic, patients will be evaluated by a Neurologist, PT, OT, social worker and coordinated appointments can be made for the same day as needed to see the Physiatrist, Orthopedist, Cardiologist, Pulmonologist, and nutritionist, all within the Institute or across the street at John Hopkins Med. Ctr.
Kennedy Krieger Institute (http://www.kennedykrieger.org/index.jsp) is affiliated to John Hopkins University, just across the street from the JH Medical Center. It is an internationally recognized facility for children with disabilities and genetic disorders. It recently opened a new building where the Muscle Clinic is located...it is amazing!! It has a therapy garden with all kind of different surfaces for evaluation of ambulation and other outdoor life activities, a kitchen and bathroom for OT and PT evaluations, a fantastic Aquatic Therapy pool with underwater treadmills and video capacity, so you can take a DVD with you to show your local PTs what you did and needs to be worked out. It also has a supervised waiting room, with a Wii, easy access and valet parking for patients, an education room and more.
Dr. Kathryn Wagner (full time) and myself (part time) will be seeing patients. We are also committed to bringing exciting clinical trials for our patients to participate, while we continue to strive in delivering aggressive diagnosis and treatment for neuromuscular disorders. As both, Dr. Wagner and myself are adult neurologists, and the Institute has broaden its patients population, we will also evaluate and follow up adult patients (no age limit to 18 or 21!), which makes a great transition place for our patients.
Although I am not in the "system" yet, you can call Jean Smoot at 443-923-9412, have them ask for an appointment in the Muscle Clinic and make a note that it is to see Dr. Escolar whenever I start my first clinic. Then, as soon as they know my starting clinic date, the will call patients in the order they have registered in this list and will start filling up the slots. I will be seeing 4 to 5 patients on a half day on Fridays, once a month. As many of you have already asked, I know the first couple of months will quickly fill up. But if we need to, I can add a second clinic day per month.
In the mean time, you can start gathering copies of your records, especially neuromuscular evaluations, genetic testing, muscle biopsies, to bring with you on the first day.
I have missed you and am happy we might be back in touch.
With warm regards,Diana Escolar
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This particular trial of ACE-031 is for those 4 and older with confirmed DMD diagnosis. While it does all you say, it completes the code for those need exon 51 to be skipped. There is also a trial for it with premenopausal women. Just nice to have three treatments to look forward to - instead of nothing - the case for so many years. I feel really good about all of this. None of these are cures and they were never professed to be, but a plausible treatment to get our kids to the next step.
Michelle said:http://www.acceleronpharma.com/content/products/ace-031.jsp
ACE-031 isn't just for DMD. It's a muscle building compound for muscle wasting diseases in general. It doesn't cure DMD or assume to. It just builds muscle. In our case, it's more of a race against time. Build muscle in the hopes of delaying the tissue degeneration.
Karen flor said:Hi! I have read many things about both the Prosena {exon skipping 51} and ACE-031 but need help!! Comparing the two, what are the advantages of one over the other or are there???? From what I understand ACE-031 can be used for a wider scope of boys than exon skipping 51. However, my son fits into the exon skipping 51 group and I'm hoping to get him into a trial, but would like to know if I'm barking up the wrong tree and the ACE-031 might be a better treatment. Any thoughts???
Karen
-
-
Awesome!!! We all just love her and are thrilled for her to be back! She will help us kick DMD's butt!
Tina said:Hi Karen. Dr. Escolar sent me this email a while back. I'm sure she wouldn't mind it if I share it with this group. Here is where you can find her. Best of luck.
Tina
Dear Families, Friends and Colleagues,
I've signed a contract with Kennedy Krieger Institute in Baltimore and will start seeing patients again on a part time basis at their new Center for Inherited Muscle Disorders. Depending on how long it takes to obtain my Maryland license, my first clinic will be once a month (on Fridays) starting probably late November or December.
Dr. Kathryn Wagner is the director of the Center, and has started seeing patients in July. At this new Muscle Clinic, patients will be evaluated by a Neurologist, PT, OT, social worker and coordinated appointments can be made for the same day as needed to see the Physiatrist, Orthopedist, Cardiologist, Pulmonologist, and nutritionist, all within the Institute or across the street at John Hopkins Med. Ctr.
Kennedy Krieger Institute (http://www.kennedykrieger.org/index.jsp) is affiliated to John Hopkins University, just across the street from the JH Medical Center. It is an internationally recognized facility for children with disabilities and genetic disorders. It recently opened a new building where the Muscle Clinic is located...it is amazing!! It has a therapy garden with all kind of different surfaces for evaluation of ambulation and other outdoor life activities, a kitchen and bathroom for OT and PT evaluations, a fantastic Aquatic Therapy pool with underwater treadmills and video capacity, so you can take a DVD with you to show your local PTs what you did and needs to be worked out. It also has a supervised waiting room, with a Wii, easy access and valet parking for patients, an education room and more.
Dr. Kathryn Wagner (full time) and myself (part time) will be seeing patients. We are also committed to bringing exciting clinical trials for our patients to participate, while we continue to strive in delivering aggressive diagnosis and treatment for neuromuscular disorders. As both, Dr. Wagner and myself are adult neurologists, and the Institute has broaden its patients population, we will also evaluate and follow up adult patients (no age limit to 18 or 21!), which makes a great transition place for our patients.
Although I am not in the "system" yet, you can call Jean Smoot at 443-923-9412, have them ask for an appointment in the Muscle Clinic and make a note that it is to see Dr. Escolar whenever I start my first clinic. Then, as soon as they know my starting clinic date, the will call patients in the order they have registered in this list and will start filling up the slots. I will be seeing 4 to 5 patients on a half day on Fridays, once a month. As many of you have already asked, I know the first couple of months will quickly fill up. But if we need to, I can add a second clinic day per month.
In the mean time, you can start gathering copies of your records, especially neuromuscular evaluations, genetic testing, muscle biopsies, to bring with you on the first day.
I have missed you and am happy we might be back in touch.
With warm regards,Diana Escolar
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